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Positive long-term Phase 1a/1b data presented at the International Symposium on Amyloidosis (ISA) 2020 demonstrate prolonged overall survival (78% at 37 months) and durable organ response –

– Phase 2 study met primary objective, supporting initiation of two parallel Phase 3 studies that will enroll ~370 AL amyloidosis patients –

BOSTON & BORDENTOWN, N.J. – SEPTEMBER 14, 2020 – Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Caelum Biosciences today announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis. The CARES clinical program includes two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease – and will collectively enroll approximately 370 patients globally. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival.   “In AL amyloidosis, misfolded amyloid proteins can build up in many organs throughout the body, including the heart and kidneys, causing significant damage to these organs and impairing their function. While current treatments address the bone marrow disorder that creates the misfolded amyloid proteins, there are no approved therapies for the significant organ damage the disease causes,” said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. “CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from these organs. Data from Phase 1 studies suggest that this treatment approach may improve organ function and long-term survival. We look forward to investigating this further in the Phase 3 clinical program.” “AL amyloidosis is particularly devastating when it affects the heart, with median survival in these patients of less than one year following diagnosis,” said Michael Spector, President and Chief Executive Officer of Caelum. “Long-term survival data from AL amyloidosis patients treated with CAEL-101 in the Phase 1a/1b study showed that 78 percent were still alive after a median follow-up time of more than three years. We recognize the urgent need for new treatments that address the organ damage caused by AL amyloidosis and are working together with the AL amyloidosis community and Alexion to advance the Phase 3 clinical program as quickly as possible.”

About the CARES Phase 3 Clinical Program
The CARES clinical program consists of two parallel double-blind, randomized, event-driven global Phase 3 studies, which are evaluating the efficacy and safety of CAEL-101 in AL amyloidosis patients who are newly diagnosed and naïve to standard of care (SoC) treatment (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy). One study is enrolling approximately 260 patients with Mayo stage IIIa disease and one study is enrolling approximately 110 patients with Mayo stage IIIb disease. The studies will be conducted at approximately 70 sites across North America, the United Kingdom, Europe, Israel, Japan, and Australia. In each study, participants are being randomized in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks. The primary study objectives are overall survival and the safety and tolerability of CAEL-101. Key secondary objectives will assess functional improvement in the six-minute walk test (6MWT), quality of life measures (Kansas City Cardiomyopathy Questionnaire Overall Score & Short Form 36 version 2 Physical Component Score) and cardiac improvement (Global Longitudinal Strain, or GLS).

Phase 2 Study Results
The Phase 2 open-label dose escalation study was conducted to investigate higher doses of CAEL-101 than had been evaluated in Phase 1 studies with a primary objective to identify the best dose to advance into Phase 3 development. The study evaluated the safety and tolerability of CAEL-101 in 13 AL amyloidosis patients at three study sites who received up to 1000 mg/m2 of CAEL-101 (two times the Phase 1 dose) administered in combination with SoC treatment. The study met its primary objectives, supporting the safety and tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose for the Phase 3 study.

Phase 1a/1b Long-Term Follow-Up Results Presented at ISA 2020
As previously reported, the Phase 1a/1b study of CAEL-101 was the first clinical trial to demonstrate improvement in cardiac function via GLS after treatment with an amyloid fibril targeted therapy in AL amyloidosis patients with amyloid cardiac involvement. New long-term follow-up data from the Phase 1a/1b study will be presented at the virtual International Symposium on Amyloidosis (ISA), September 14 to 18, 2020, in the poster titled, “Long term follow-up of patients with AL amyloidosis treated on a phase 1 study of Anti-Amyloid Monoclonal Antibody CAEL-101” (Abstract #342, Divaya Bhutani, M.D., et. al, Columbia University Medical Center). These data demonstrate 78 percent survival (15/19) at a median follow-up of more than three years (37 months) in AL amyloidosis patients treated with CAEL-101 as well as durable organ response among evaluable patients, further supporting the advancement of CAEL-101 into Phase 3 development.

About CAEL-101
CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a therapy for patients with AL amyloidosis.

About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant. AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About Alexion
Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com. [ALXN-P]

About Caelum Biosciences
Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. In 2019, Caelum entered a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Caelum was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit www.caelumbio.com.

Forward-Looking Statement
This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion and Caelum, including statements related to: the safety and efficacy CAEL-101 as a treatment for AL amyloidosis; CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from the heart, kidney and other organs; data from the Phase 1 studies suggest that the treatment approach may improve organ function and long-term survival and enrollment of the Phase 3 trials. Forward-looking statements are subject to factors that may cause Alexion’s and Caelum’s results and plans to differ materially from those expected by these forward looking statements, including for example: the anticipated safety profile and the benefits of the CAEL-101 may not be realized (and the results of the clinical trials may not be indicative of future results); the inability to enroll and complete the Phase 3 trial; results of clinical trials may not be sufficient to satisfy regulatory authorities; results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexion’s or Caelum’s business, including on commercial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding our products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us; the risk that third party payors (including governmental agencies) will not reimburse for the use of our products at acceptable rates or at all; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including COVID-19 and a variety of other risks set forth from time to time in Alexion’s filings with the SEC, including but not limited to the risks discussed in Alexion’s Quarterly Report on Form 10-Q for the period ended June 30, 2020 and in their other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

Alexion Contacts:
Media
Megan Goulart, 857-338-8634
Executive Director, Corporate Communications

Investors
Chris Stevo, 857-338-9309
Head of Investor Relations

Caelum Contacts:
Company
Michael Spector, President & Chief Executive Officer
mspector@caelumbio.com

Jaclyn Jaffe and William Begien
Investor Relations
(781) 652-4500
info@caelumbio.com

Media
Tony Plohoros
6 Degrees
(908) 591-2839
tplohoros@6degreespr.com

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BORDENTOWN, NJ – December 2, 2019 – Caelum Biosciences, Inc. (“Caelum”), a company focused on developing treatments for rare and life-threatening diseases, today announced that the European Commission (EC) has granted orphan medicinal product designation to CAEL-101 (previously known as 11-1F4), a light chain fibril-reactive monoclonal antibody (“mAb”), for the treatment of amyloid light chain (“AL”) amyloidosis.  CAEL-101 is a first-in-class amyloid fibril targeted therapy designed to improve organ function by reducing or eliminating amyloid deposits in patients with AL amyloidosis. AL amyloidosis is a rare systemic disorder that causes misfolded immunoglobulin light chain protein to build up in and around tissues, resulting in progressive and widespread organ damage, most commonly to the heart and kidneys.  CAEL-101 previously received orphan drug designation (“ODD”) from the U.S. Food and Drug Administration (“FDA”) as a therapy for patients with AL amyloidosis, and as a radio-imaging agent in AL amyloidosis.

Orphan Medicinal Product Designation in the European Union (“EU”) is based upon a positive opinion from the European Medicines Agency’s (“EMA”) Committee for Orphan Medicinal Products and provides regulatory and financial incentives for companies to develop and market therapies to treat serious disorders affecting no more than five in 10,000 persons in the EU. Companies that obtain orphan medicinal product designation benefit from a number of incentives, including ten-year marketing exclusivity in the EU upon approval, as well as eligibility for protocol assistance, reduced fees and access to the EU’s centralized marketing authorization procedure.

About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs, gradually affecting their function. This can cause progressive and widespread organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant. 

AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About CAEL-101 (mAb 11-1F4)
CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to insoluble light chain amyloid protein, including both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapy for patients with AL amyloidosis, and as a radio-imaging agent in AL amyloidosis.

About Caelum Biosciences
Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. For more information, visit www.caelumbio.com.

Forward-Looking Statements
This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contact:
Caelum Biosciences, Inc.
Michael Spector, President & Chief Executive Officer
mspector@caelumbio.com

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– Collaboration provides opportunity to diversify Alexion’s clinical-stage rare hematology portfolio –

– Caelum to receive up to $60 million for equity investment and option fee –

– Alexion has option to acquire Caelum based on Phase 2 data –

BOSTON & NEW YORK–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) and Caelum Biosciences today announced a collaboration to develop CAEL-101 for light chain (AL) amyloidosis. CAEL-101 is a first-in-class amyloid fibril targeted therapy designed to improve organ function by reducing or eliminating amyloid deposits in patients with AL amyloidosis. AL amyloidosis is a rare systemic disorder that causes misfolded immunoglobulin light chain protein to build up in and around tissues, resulting in progressive and widespread organ damage, most commonly to the heart and kidneys.

With a median survival time of less than 18 months following diagnosis and no approved therapies to address the organ damage caused by AL amyloidosis, there is a significant need for new treatments for this devastating disease,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “We believe CAEL-101 holds significant promise in being able to help these patients and we’re excited to add it to our growing clinical-stage rare hematology portfolio.”

Michael Spector, President and Chief Executive Officer of Caelum, said, “CAEL-101 appears to have a unique capability of binding to both kappa and lambda misfolded proteins. Data from the Phase 1a/1b study indicate that CAEL-101 is a well-tolerated therapy that leads to a rapid and clinically relevant organ response, particularly in the heart and kidneys. Further, CAEL-101 showed a statistically significant improvement from baseline in global longitudinal strain, an endpoint that has been correlated with survival in patients with AL amyloidosis. We are very pleased to collaborate with Alexion, a global leader in the rare disease field.”

Under the terms of the agreement, Alexion will acquire a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 2 data for pre-negotiated economics. Alexion will make payments to Caelum totaling up to $60 million, including the purchase price for the equity and milestone-dependent development funding payments. The collaboration also provides for potential additional payments of up to $500 million, including the upfront and regulatory and commercial milestone payments, in the event Alexion exercises the acquisition option. The collaboration will leverage Alexion’s expertise in rare disease antibody development and commercial franchise in hematology. Alexion and Caelum will collaborate on the design of the ongoing development program for CAEL-101. Caelum will be responsible for conducting the development program through the end of Phase 2 and for manufacturing CAEL-101.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to insoluble light chain amyloid protein, including both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapy for patients with AL amyloidosis and as a radio-imaging agent in AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs, gradually affecting their function. This can cause progressive and widespread organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH), as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS) and anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG), and is also developing it for patients with neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology and metabolic disorders. Alexion has been named to the Forbes list of the World’s Most Innovative Companies seven years in a row and is headquartered in Boston, Massachusetts’ Innovation District. The company also has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.

[ALXN-G]

About Caelum Biosciences

Caelum Biosciences, founded by Fortress Biotech, Inc.(NASDAQ: FBIO) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. Phase 1a/1b data support CAEL-101’s potential to be a well-tolerated therapy that promotes amyloid resolution. For more information, visit www.CaelumBio.com.

Forward-Looking Statement

This press release includes forward-looking statements, including statements related to the therapeutic benefits of CAEL-101, the potential of CAEL-101 as a treatment for AL amyloidosis and the potential benefits of the collaboration. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. The process by which an early stage product such as CAEL-101 could potentially lead to an approved product for the treatment of AL amyloidosis is long and subject to highly significant risks, including for example, decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of products, delays, interruptions or failures in manufacture and supply, failure to satisfactorily address matters raised by the U.S. Food and Drug Administration and other regulatory agencies, the possibility that results of clinical trials are not predictive of safety and efficacy results in broader patient populations, the possibility that clinical trials could be delayed, the risk that anticipated regulatory filings are delayed, the risk that estimates regarding the number of patients with AL amyloidosis are inaccurate, and a variety of other risks set forth from time to time in Alexion’s filings with the SEC, including but not limited to the risks discussed in Alexion’s Quarterly Report on Form 10-Q for the period ended September 30, 2018 and in Alexion’s other filings with the SEC. Alexion and Caelum disclaim any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

Contacts

Alexion:
Media
Megan Goulart, 857-338-8634
Senior Director, Corporate Communications

Investors
Susan Altschuller, Ph.D., 857-338-8788
Vice President, Investor Relations

Caelum:
Michael Spector, 609-845-7088
President & Chief Executive Officer
mspector@caelumbio.com

Media
Tony Plohoros, 908-940-0135
6 Degrees
tplohoros@6degreesPR.com

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Phase 1b data confirm efficacy in AL Amyloidosis with two cardiac markers correlated with survival

 Preclinical data demonstrate the potential of using radiolabeled CAEL-101 for real-time imaging of human amyloidosis in vivo

 NEW YORK, NY – December 4, 2018 – Caelum Biosciences, Inc. (“Caelum”), a company focused on developing treatments for rare and life-threatening diseases, today announced additional global longitudinal strain (“GLS”) data from the Phase 1b study of CAEL-101, a light chain fibril-reactive monoclonal antibody (“mAb”) 11-1F4, in patients with cardiac amyloid light chain (“AL”) amyloidosis, which further confirmed CAEL-101’s efficiency improving GLS and NT-proBNP. The Company also announced imaging data from a preclinical study that demonstrate the potential of using radiolabeled CAEL-101 for real-time imaging of human amyloidosis in vivo. The data were presented during two oral sessions at the 60th American Society of Hematology (“ASH”) Annual Meeting by Divaya Bhutani, M.D., and Jing Fu, Ph.D., of Columbia University Medical Center (“CUMC”).

In the Phase 1b study of relapsed and refractory systemic AL amyloidosis, 10 out of 19 patients had cardiac involvement, with a median NT-proBNP of 1186 (range 699-3964) at screening. CAEL-101 was administered weekly for four weeks with sequential doses of 0.5, 5, 10, 50, 100, 250 and 500 mg/m2 in a dose-escalation design. All patients underwent transthoracic echocardiograms at screening and 12 weeks post-therapy.

Eight of 10 patients who had cardiac involvement were considered evaluable for NT-proBNP response. Six out of the eight patients (75%) with cardiac involvement met cardiac response criteria by having a decrease in NT-proBNP 30%. Among echocardiographic parameters, mean GLS improved significantly in nine out of 10 patients from -15.58 ± -4.14% at screening to -17.37 ± -3.53% at week 12, p = 0.004 of the trial. Four out of seven patients (57%) with renal involvement met renal response criteria with ≥ 50% decrease in 24-hour urine proteinuria.

Median time to organ response was three weeks. There was no change in patients without cardiac involvement. Seventeen of 19 patients studied were alive at follow up of 19 months.

“We are pleased that these data demonstrate CAEL-101’s ability to induce rapid organ responses in patients with AL amyloidosis. We also found that improvement in GLS corresponds with improvement in NT-proBNP in patients with cardiac AL amyloidosis treated with CAEL-101. It is important to note that GLS is a sensitive measure of left ventricular function that is correlated with survival in AL amyloidosis,” said Dr. Bhutani, Assistant Professor of Medicine at CUMC.

The preclinical study explored the diagnostic potential of CAEL-101 radiolabeled with a positron emitting radioisotope for systemic amyloidosis and its use as a companion biomarker to stratify patients for CAEL-101 immunotherapy. CAEL-101 successfully imaged 100% of mice bearing human amyloid extracts of both kappa and lambda subtypes derived from the heart, spleen, liver and kidney. These findings indicate that using CAEL-101 with positron emission tomography (PET) imaging may facilitate diagnosis of systemic amyloidosis, stratify patients for CAEL-101 immunotherapy and quantify peripheral organ amyloid fibril deposition pre- and post-anti-amyloid therapy. The study of mAb CAEL-101 confirmed the findings of previous studies with the murine monoclonal antibody, which was evaluated in preclinical and clinical studies.

“We successfully used PET imaging to visualize cardiac-derived amyloid fibrils from AL amyloidosis patients, with clinically significant imaging of both kappa and lambda fibrils. Because heart failure is the most critical condition that can impede AL amyloidosis patients’ survival, successfully conducting in vivo imaging of cardiac amyloidosis is essential for diagnostic purposes,” said Dr. Fu, Associate Research Scientist at CUMC. “As a result of our findings, we anticipate that dedicated gated cardiac PET and computerized tomography (CT) imaging of radiolabeled CAEL-101 will be successful in visualizing cardiac amyloid deposits in patients, especially with the rich blood flow in cardiac tissue and newer-generation, highly sensitive, high-resolution digital PET scanners.”

About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells cause buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage and high mortality rates.

AL amyloidosis affects roughly 30,000 – 40,000 patients in total throughout the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the U.S., though actual incidence is likely higher as a result of under-diagnosis. Amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

 About CAEL-101 (mAb 11-1F4)
CAEL-101 is a light chain fibril-reactive monoclonal antibody (mAb) that has completed a Phase 1a/1b trial at Columbia University for the treatment of patients with AL amyloidosis. While current treatment with chemotherapy is aimed at reducing production of the amyloid-forming light-chain protein, CAEL-101 attempts to reduce and / or eliminate the amyloid deposits.

 About Caelum Biosciences
Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. Phase 1a/1b data support CAEL-101’s potential to be a safe and well-tolerated therapy that promotes amyloid resolution. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio-imaging agent in amyloidosis. For more information, visit www.caelumbio.com.

 Forward-Looking Statements
This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contacts:
Caelum Biosciences, Inc.
Michael Spector, President & Chief Executive Officer
(212) 574-2811
mspector@caelumbio.com

Media Relations
Tony Plohoros
6 Degrees
(908) 940-0135
tplohoros@6degreesPR.com

 

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NEW YORK, NY – November 2, 2018 – Caelum Biosciences, Inc. (“Caelum”), a company focused on developing treatments for rare and life-threatening diseases, today announced that additional global longitudinal strain (“GLS”) data from the Phase 1b study of CAEL-101 (a light chain fibril-reactive monoclonal antibody 11-1F4) in patients with amyloid light chain (“AL”) amyloidosis and imaging data from a pre-clinical study have been selected for two oral presentations at the 60th American Society of Hematology (“ASH”) Annual Meeting, to be held December 1-4, 2018, in San Diego, CA.

Details of the oral presentations are as follows:

Title: Improvement in Global Longitudinal Strain (GLS) Correlates with NT-ProBNP Response in Patients with Cardiac Amyloidosis Treated on a Phase 1b Study of Anti-Amyloid mAb CAEL-101
Abstract Number: 958
Oral Session: 653. Myeloma: Therapy, Excluding Transplantation: Immunotherapies in Plasma Cell Disorders
Date and Time: Monday, December 3, 2018; 4:30 PM – 6:00 PM PT
Presentation Time: 5:15 PM PT
Location: San Diego Convention Center, Ballroom 20A
Presenter: Divaya Bhutani, M.D., Columbia University Medical Center, New York, NY

Title: Personalizing Amyloidosis Therapy with Real Time PET Imaging of Fibril-Reactive Monoclonal Antibody CAEL-101
Abstract Number: 1003
Oral Session: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, Excluding Therapy: Molecular Mechanisms of Myelomagenesis and Its Dependencies
Date and Time:  Monday, December 3, 2018; 6:15 PM – 7:45 PM PT
Presentation Time: 6:15 PM PT
Location: San Diego Convention Center, Ballroom 20A
Presenter: Jing Fu, Ph.D., Columbia University Medical Center, New York, NY

The abstracts can be viewed online through the ASH meeting website at www.hematology.org.

About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells cause buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage and high mortality rates.

AL amyloidosis affects roughly 30,000 – 40,000 patients in total throughout the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the U.S., though actual incidence is likely higher as a result of under-diagnosis. Amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About CAEL-101 (mAb 11-1F4)
CAEL-101 is a light chain fibril-reactive monoclonal antibody (mAb) that has completed a Phase 1a/1b trial at Columbia University for the treatment of patients with AL amyloidosis. While current treatment with chemotherapy is aimed at reducing production of the amyloid-forming light-chain protein, CAEL-101 attempts to reduce and / or eliminate the amyloid deposits.

 About Caelum Biosciences
Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. Phase 1a/1b data support CAEL-101’s potential to be a safe and well-tolerated therapy that promotes amyloid resolution. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio-imaging agent in amyloidosis. For more information, visit www.caelumbio.com.

Forward-Looking Statements
This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contacts:
Caelum Biosciences, Inc.
Michael Spector, President & Chief Executive Officer
(212) 574-2811
mspector@caelumbio.com

Media Relations
Tony Plohoros
6 Degrees
(908) 940-0135
tplohoros@6degreespr.com

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Nine of 10 cardiac patients on CAEL-101 showed improvement in global longitudinal strain  

 CAEL-101 led to sustained decrease in NT-proBNP levels

 Data presented at American Society of Echocardiography 29th Annual Scientific Sessions

NEW YORK, NY – June 25, 2018 – Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech, Inc. (NASDAQ: FBIO) Company developing treatments for rare and life-threatening diseases, today announced a complete analysis of cardiac data from Columbia University’s (“Columbia”) Phase 1b trial that supports CAEL-101’s (mAb 11-1F4) potential to improve myocardial function as assessed by global longitudinal strain (“GLS”) and generate a sustained decrease in N-terminal pro-brain natriuretic peptide (NT-proBNP) levels in amyloid light chain (“AL”) amyloidosis patients experiencing cardiac involvement. These data were presented at the American Society of Echocardiography (ASE) 29th Annual Scientific Sessions.

“Cardiac involvement can indicate a poor prognosis in patients with AL amyloidosis, and is the cause of more than 75 percent of one-year mortality,” said Sofia Shames, M.D., Assistant Professor at Columbia University Medical Center and investigator on the Phase 1b trial. “The Phase 1b trial of CAEL-101 is the first clinical study to show a significant improvement in GLS after exposure to an anti-fibril specific mAb in people with AL amyloid cardiac involvement. This is important, as echocardiographical GLS may be more effective in predicting overall and cardiovascular mortality than traditional ejection fraction, which warrants further evaluation in clinical trials.”

Myocardial response was assessed using GLS as part of a long-term analysis of data from the Phase 1b trial. GLS is a measure of myocardial shortening during systole, which may detect early cardiac functional improvement and be a predictor of cardiac survival.

Nineteen patients with relapsed or refractory AL amyloidosis enrolled in the Phase 1b trial had a baseline echocardiogram taken at their initial screening visit. Ten of 19 patients (52 percent) had cardiac involvement at the time of their baseline echocardiogram reading as defined by NT-proBNP levels (>650 pg/ml), an important biomarker in cardiac disease. CAEL-101 was administered weekly for four weeks in the multi-ascending dose trial. The dose escalation included the doses 0.5, 5, 10, 50, 100, 250 and 500 mg/m2. Clinical echocardiographic examinations at baseline and 12 weeks post therapy were compared.

Patients with cardiac involvement demonstrated an improvement in GLS (-15.58 ± -4.14 percent pre-treatment / -17.37 ± -3.53 percent post-treatment, p=0.004). Patients with both kappa and lambda amyloid light chain subtypes were studied. The change in GLS patients with a p-value of 0.004 and the lack of response in patients without cardiac involvement supports further clinical development in patients with cardiac AL amyloidosis.

In addition, mean NT-proBNP reduction was presented, which demonstrated that there was an improvement in NT-proBNP in eight evaluable patients with cardiac amyloid involvement after four weekly doses. The NT-proBNP patient response criteria were a 30-percent reduction and a 300 pg/ml reduction from baseline.

The full abstract was published in the June 2018 edition of the Journal of the American Society of Echocardiography.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells cause buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage, and high mortality rates.

AL amyloidosis affects roughly 30,000 – 40,000 patients in total throughout the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the U.S., though actual incidence is likely higher as a result of under-diagnosis. Amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

 About CAEL-101 (mAb 11-1F4)

CAEL-101 is a chimeric fibril-reactive monoclonal antibody (mAb) that has completed a Phase 1a/1b trial at Columbia University for the treatment of patients with relapsed or refractory AL amyloidosis. While current treatment with chemotherapy is aimed at reducing production of the amyloid-forming light-chain protein, CAEL-101 attempts to reduce and / or eliminate the amyloid deposits.

About the Phase 1a/1b trial

The Phase 1a/1b trial (ClinicalTrials.gov Identifier: NCT02245867) examined the tolerance, safety, pharmacokinetics and possible clinical benefit of CAEL-101 (mAb 11-1F4) in patients with relapsed or refractory AL amyloidosis. CAEL-101 was administered weekly for four weeks with sequential doses of 0.5, 5, 10, 50, 100, 250 and 500 mg/m2. CAEL-101 was administered to eight patients via a single IV infusion at week one in the Phase 1a portion of the trial, and to 19 patients via one weekly IV infusion for four weeks in the Phase 1b portion of the trial.

About Caelum Biosciences

Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech (NASDAQ: FBIO) Company, is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. Phase 1a/1b data presented at the American Society of Hematology’s 59th Annual Meeting in December 2017 support CAEL-101’s potential to be a safe and well-tolerated therapy that promotes amyloid resolution. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio-imaging agent in amyloidosis. For more information, visit www.caelumbio.com.

About Fortress Biotech

Fortress Biotech, Inc. (“Fortress”) is a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products. Fortress develops and commercializes products both within Fortress and through certain subsidiary companies, also known as Fortress Companies. In addition to its internal development programs, Fortress leverages its biopharmaceutical business expertise and drug development capabilities and provides funding and management services to help the Fortress Companies achieve their goals. Fortress and the Fortress Companies may seek licensings, acquisitions, partnerships, joint ventures and/or public and private financings to accelerate and provide additional funding to support their research and development programs. For more information, visit www.fortressbiotech.com.

Forward-Looking Statements

This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contacts:
Caelum Biosciences, Inc.
Michael Spector, President & Chief Executive Officer
(212) 574-2811
mspector@caelumbio.com

Fortress Biotech, Inc.
Jaclyn Jaffe, Investor Relations
(781) 652-4500
ir@fortressbiotech.com

Fortress Biotech, Inc.
Tony Plohoros, Media Relations
6 Degrees
(908) 591-2839
tplohoros@6degreesPR.com

 

Category Archives: Uncategorized

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Columbia University to present data demonstrating CAEL-101 positively impacts myocardial function in patients with AL amyloidosis as measured by global longitudinal strain

NEW YORK, NY – June 18, 2018 – Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech, Inc. (NASDAQ: FBIO) Company developing treatments for rare and life-threatening diseases, today announced that Columbia University (“Columbia”) will present a complete analysis of cardiac data from the Phase 1b trial of CAEL-101 (mAb 11-1F4) for the treatment of relapsed or refractory amyloid light chain (“AL”) amyloidosis in a poster session at the American Society of Echocardiography (ASE) 29th Annual Scientific Sessions, to be held June 22-26, 2018, in Nashville, Tenn.

Investigators concluded that the Phase 1b trial of CAEL-101 in AL amyloidosis is the first trial to show a significant improvement in global longitudinal strain (“GLS”) after exposure to an anti-fibril specific mAb in subjects with AL amyloidosis experiencing cardiac involvement. GLS is a sensitive measure of cardiac functional impairment in patients with cardiac involvement, and may detect early cardiac functional improvement and be an important predictor of cardiac survival.

Details of the presentation are as follows:

Title: Cardiac Response to Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients with AL Amyloidosis with Global Longitudinal Strain: Results from the Phase 1b trial
Session: Poster Session 1
Poster Number: P1-01
Date and Time: Sunday, June 24, 2018, from 9 a.m. to 4 p.m. CDT
Location: Ryman Exhibit Hall, lower level of the Gaylord Opryland Resort & Convention Center
Presenter: Sofia Shames, M.D., Columbia University Medical Center

The full abstract was published in the June 2018 edition of the Journal of the American Society of Echocardiography. For more information on ASE, please visit http://asescientificsessions.org/.

About Caelum Biosciences

Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech (NASDAQ: FBIO) Company, is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. Phase 1a/1b data presented at the American Society of Hematology’s 59th Annual Meeting in December 2017 support CAEL-101’s potential to be a safe and well-tolerated therapy that promotes amyloid resolution. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio-imaging agent in amyloidosis. For more information, visit www.caelumbio.com.

About Fortress Biotech

Fortress Biotech, Inc. (“Fortress”) is a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products. Fortress develops and commercializes products both within Fortress and through certain subsidiary companies, also known as Fortress Companies. In addition to its internal development programs, Fortress leverages its biopharmaceutical business expertise and drug development capabilities and provides funding and management services to help the Fortress Companies achieve their goals. Fortress and the Fortress Companies may seek licensings, acquisitions, partnerships, joint ventures and/or public and private financings to accelerate and provide additional funding to support their research and development programs. For more information, visit www.fortressbiotech.com.

Forward-Looking Statements

This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contacts:

Caelum Biosciences, Inc.
Michael Spector, President & Chief Executive Officer
(212) 574-2811
mspector@caelumbio.com

Fortress Biotech, Inc.
Jaclyn Jaffe, Investor Relations
(781) 652-4500
ir@fortressbiotech.com

Fortress Biotech, Inc.
Laura Bagby, Media Relations
6 Degrees
(312) 448-8098
lbagby@6degreespr.com

Category Archives: Uncategorized

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NEW YORK, NY – April 20, 2018 – Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech, Inc. Company developing treatments for rare and life-threatening diseases, today announced it has confidentially submitted a draft registration statement on Form S-1 to the Securities and Exchange Commission (the “SEC”) related to its proposed initial public offering. The number of shares of stock and the price range for the proposed offering have not yet been determined. The proposed offering is subject to, among other things, completion of SEC review process and market conditions.

This press release is being made pursuant to and in accordance with Rule 135 under the Securities Act of 1933, as amended, and does not constitute an offer to sell or the solicitation of an offer to buy securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction.

Contacts:
Caelum Biosciences, Inc.
Michael Spector, President & Chief Executive Officer
(212) 574-2811
mspector@caelumbio.com

Laura Bagby, Media Relations
6 Degrees
(312) 448-8098
lbagby@6degreespr.com

Category Archives: Uncategorized

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New data analysis demonstrates correlation between sustained decrease in NT-proBNP and improvement in global longitudinal strain in cardiac population following CAEL-101 treatment

NEW YORK, NY – March 27, 2018 – Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech, Inc. (NASDAQ: FBIO) Company developing treatments for rare and life-threatening diseases, today announced a new analysis of data from the Phase 1b trial of CAEL-101 (mAb 11-1F4) for the treatment of relapsed or refractory amyloid light chain (“AL”) amyloidosis, demonstrating a correlation between a sustained decrease in N-terminal pro-brain natriuretic peptide (NT-proBNP) levels and an improvement in global longitudinal strain (GLS) following CAEL-101 treatment in patients with cardiac AL amyloidosis. The data were presented today in an oral session at the 16th International Symposium on Amyloidosis (ISA).

The Phase 1a/1b trial (ClinicalTrials.gov Identifier: NCT02245867) examined the tolerance, safety, pharmacokinetics and possible clinical benefit of CAEL-101 in patients with AL amyloidosis. CAEL-101 was administered to eight patients via a single IV infusion at week one in the Phase 1a portion of the trial, and to 19 patients via one weekly IV infusion for four weeks in the Phase 1b portion of the trial.

Eight of 12 (67 percent) of evaluable cardiac patients in the Phase 1a/1b trial demonstrated a sustained decrease in NT-proBNP levels, an important biomarker in cardiac disease, after treatment with CAEL-101. Evaluable patients presented with cardiac amyloidosis at baseline (NT-proBNP ≥650 pg/mL) and at least one post-baseline NT-proBNP measure. The depth and magnitude of response continued through four once-weekly doses of CAEL-101.

An improvement in GLS, a measure of myocardial shortening during systole, was demonstrated in eight patients with confirmed cardiac amyloidosis at baseline who were enrolled in the Phase 1b portion of the trial, and was correlated with the reduction in NT-proBNP (Pearson correlation coefficient[i] 0.345), demonstrating CAEL-101’s positive impact on cardiac response of myocardial function.

“GLS is an important biomarker for cardiac dysfunction and response, and may be a more accurate predictor of survival than cardiac biomarkers, which can be elevated disproportionately to the severity of cardiac symptoms and based on factors unrelated to worsening cardiac dysfunction,” said Camille Edwards, M.D., Fellow in the Department of Hematology and Oncology at Boston Medical Center, who presented the data at ISA. “CAEL-101 is a novel and promising treatment that has the potential to safely promote amyloid resolution and improve cardiac and overall organ function in AL amyloidosis. The presentation of a complete analysis of cardiac data from the Phase 1b trial is planned for later this year.”

“The correlation of GLS improvement with NT-proBNP reduction builds upon the strong Phase 1a/1b safety and efficacy data presented at ASH 2017, and it is important to note that efficacy was demonstrated independent of plasma-cell directed therapy,” said Michael Spector, President and Chief Executive Officer of Caelum.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells cause buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage, and high mortality rates.

AL amyloidosis affects roughly 30,000 – 40,000 patients in total throughout the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the U.S., though actual incidence is likely higher as a result of under diagnosis. Amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About CAEL-101 (mAb 11-1F4)

CAEL-101 is a chimeric fibril-reactive monoclonal antibody (mAb) that has completed a Phase 1a/1b clinical trial at Columbia University for the treatment of patients with AL amyloidosis. While current treatment with chemotherapy is aimed at reducing production of the amyloid-forming light-chain protein, CAEL-101 attempts to reduce and / or eliminate the amyloid deposits.

About Caelum Biosciences

Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech (NASDAQ: FBIO) Company, is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. Phase 1a/1b data presented at the American Society of Hematology’s 59th Annual Meeting in December 2017 support CAEL-101’s potential to be a safe and well-tolerated therapy that promotes amyloid resolution. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio-imaging agent in amyloidosis. For more information, visit www.caelumbio.com.

About Fortress Biotech

Fortress Biotech, Inc. (“Fortress”) is a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products. Fortress develops and commercializes products both within Fortress and through certain subsidiary companies, also known as Fortress Companies. In addition to its internal development programs, Fortress leverages its biopharmaceutical business expertise and drug development capabilities and provides funding and management services to help the Fortress Companies achieve their goals. Fortress and the Fortress Companies may seek licensings, acquisitions, partnerships, joint ventures and/or public and private financings to accelerate and provide additional funding to support their research and development programs. For more information, visit www.fortressbiotech.com.

Forward-Looking Statements

This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

 

Contacts:

Caelum Biosciences, Inc.

Michael Spector, President & Chief Executive Officer

(212) 574-2811

mspector@caelumbio.com

 

Fortress Biotech, Inc.

Jaclyn Jaffe, Investor Relations

(781) 652-4500

ir@fortressbiotech.com

Fortress Biotech, Inc.

 

Laura Bagby, Media Relations

6 Degrees

(312) 448-8098

lbagby@6degreespr.com

 

[i] The Pearson correlation coefficient is a measure of the linear correlation between two variables.

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Final analysis of data from Phase 1a/1b trial and additional global longitudinal strain response to be presented

 NEW YORK, NY – March 21, 2018 – Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech, Inc. (NASDAQ: FBIO) Company developing treatments for rare and life-threatening diseases, today announced that the final analysis of data and additional global longitudinal strain response from the Phase 1a/1b trial of Caelum’s CAEL-101 (mAb 11-1F4) for the treatment of relapsed or refractory amyloid light chain (“AL”) amyloidosis will be presented in an oral session at the 16th International Symposium on Amyloidosis, to be held March 26-29, 2018, in Kumamoto, Japan. The Phase 1a/1b trial was conducted at Columbia University.

Details of the presentation are as follows:
Title: Final Analysis of the Phase 1a/b Study of Fibril-Reactive Monoclonal Antibody 11-1F4 (CAEL-­101) in Patients with AL Amyloidosis
Session: AL amyloidosis 3
Oral Presentation Number: 39
Date and Time: Tuesday, March 27, 2018, at 4:30 p.m. JST
Location: KKR Hotel Kumamoto
Presenter: Camille V. Edwards, M.D., Boston Medical Center, Boston

For more information, please visit isa2018.com.

About Caelum Biosciences
Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech (NASDAQ: FBIO) Company, is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. Phase 1a/1b data presented at the American Society of Hematology’s 59th Annual Meeting in December 2017 support CAEL-101’s potential to be a safe and well-tolerated therapy that promotes amyloid resolution. CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio-imaging agent in amyloidosis. For more information, visit www.caelumbio.com.

About Fortress Biotech
Fortress Biotech, Inc. (“Fortress”) is a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products. Fortress develops and commercializes products both within Fortress and through certain subsidiary companies, also known as Fortress Companies. In addition to its internal development programs, Fortress leverages its biopharmaceutical business expertise and drug development capabilities and provides funding and management services to help the Fortress Companies achieve their goals. Fortress and the Fortress Companies may seek licensings, acquisitions, partnerships, joint ventures and/or public and private financings to accelerate and provide additional funding to support their research and development programs. For more information, visit www.fortressbiotech.com.

Forward-Looking Statements
This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contacts:

Caelum Biosciences, Inc.
Michael Spector, President & Chief Executive Officer
(212) 574-2811
mspector@caelumbio.com

Fortress Biotech, Inc.
Jaclyn Jaffe, Investor Relations
(781) 652-4500
ir@fortressbiotech.com

Fortress Biotech, Inc.
Laura Bagby, Media Relations
6 Degrees
(312) 448-8098
lbagby@6degreespr.com