Bioverativ Announces First Patient Dosed in Phase 3 Study of BIVV009 for Treatment of Cold Agglutini

 Bioverativ Inc. (NASDAQ: BIVV), a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced that the first patient has been dosed in the Phase 3 clinical program of its investigational therapy BIVV009 for cold agglutinin disease (CAgD). The Phase 3 program includes two parallel Phase 3 trials, Cardinal and Cadenza, which are evaluating the efficacy and safety of BIVV009 in adult patients with primary CAgD, a disease with no approved therapies.

CAgD is a rare blood disease that results in the premature destruction of red blood cells (hemolysis) by the body’s immune system. People with CAgD suffer from anemia, which is often severe, and can experience profound fatigue, and/or a significantly increased risk of life-threatening thromboembolic (TE) events such as stroke, pulmonary embolism, and heart attack. Recently-presented results from the CAgD Optum study, the largest retrospective study of cold agglutinin disease, showed that CAgD patients had a 55% overall increased rate of TE events versus matched controls (31% vs. 20% p<0.0001), as well as a statistically significant higher frequency of multiple TE events.¹

"I am optimistic that the initiation of the Phase 3 trials of BIVV009 moves us closer to a new era of treatment for people living with cold agglutinin disease," said Catherine Broome, M.D., Associate Professor, Georgetown University. "People with cold agglutinin disease can suffer from a significant disease burden, and data from the Phase 1 study suggests that BIVV009 may be able to address the underlying hemolysis, which is at the core of the disease."

The Cardinal trial, in which the patient was dosed, is an open-label, single-arm study that will evaluate the safety and efficacy of BIVV009 in 20 adult patients with primary CAgD who have had at least one recent blood transfusion. The Cadenza trial is a randomized, double-blind, placebo-controlled study that will evaluate the safety and efficacy of BIVV009 in 40 adult patients with primary CAgD who have not recently had a blood transfusion.

"The start of our Phase 3 trials is an important step toward bringing the first approved therapy for cold agglutinin disease to patients who are greatly in need of a safe and effective treatment," said Joachim Fruebis, Senior Vice President of Development at Bioverativ. "Based on results from a Phase 1b trial, we believe that BIVV009 has the potential to meaningfully improve the lives of people with CAgD, and we are focused on quickly advancing the program."

BIVV009 is a novel monoclonal antibody that has been designed to block C1s in the classical complement pathway and directly target the root cause of cold agglutinin disease. It is believed that this approach may prevent the complement system, which is a part of the immune system, from mistakenly destroying a person’s red blood cells, and potentially halt the CAgD disease process.

BIVV009 was awarded Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation by both the FDA and European Medicines Agency. These designations acknowledge the potential of novel drugs and biologics, and are intended to expedite the development and regulatory review of such therapies.

About the BIVV009 Phase 1b Study
The Phase 1b study assessed the long-term efficacy, safety, and PK/PD profile of BIVV009 in six severely anemic primary CAgD patients. Primary and secondary outcome measures were achieved in the CAgD patients in the study. Hemoglobin levels increased in all six patients (median >4g/dl), eliminating the need for transfusions while on treatment. Maintenance therapy through a Named Patient Program has demonstrated a sustained response for more than 18 months, including control of hemolysis. Safety data through December 21, 2016, demonstrated that BIVV009 was generally well tolerated. Five of six patients (83.3%) in the primary CAgD group experienced at least one adverse event (AE); no AE was reported by more than one patient. One unrelated, serious AE occurred in a patient with CAgD who was hospitalized for a pre-existing condition. There were no serious AEs assessed as related to BIVV009 by the investigator.

About Cold Agglutinin Disease (CAgD)
CAgD is a severe, chronic rare blood disease in which a part of the body’s immune system called the complement system mistakenly attacks a person’s own red blood cells. People with CAgD suffer from chronic anemia, severe fatigue, and an increased risk of life-threatening events such as stroke. There are no approved therapies for CAgD, which occurs in approximately 16 people per million,² including an estimated 10,000 people in the United States and Europe. Current treatment options are aimed at normalizing hemoglobin levels through blood transfusions, immunomodulating therapy not approved for CAgD, or chemotherapy using cytotoxic agents. These options have significant toxicity risks and are often nondurable, leaving patients dependent on frequent transfusions, which can lead to chronic iron overload

About BIVV009
BIVV009 is a first-in-class, humanized, monoclonal antibody that has been specifically designed to target C1s, a serine protease within the C1-complex, that is the first step in activating the classical complement pathway of the immune system. The classical complement pathway is the central mechanism responsible for the development of hemolytic anemia in cold agglutinin disease and blocking it may potentially halt the CAgD disease process. With a novel mechanism of action and high target specificity, BIVV009 is designed to selectively inhibit disease processes upstream in the classical complement pathway while leaving intact the alternative and lectin complement pathways and their immune surveillance functions.

About Bioverativ
Bioverativ is a global biopharmaceutical company dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development, and commercialization of innovative therapies. Launched in 2017 following separation from Biogen Inc., Bioverativ builds upon a strong heritage of scientific innovation and is committed to actively working with the blood disorders community. The company’s mission is to create progress for patients where they need it most, and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. 

Bioverativ Safe Harbor
This press release contains forward-looking statements, including statements about the potential benefits and improvements in the lives of people with CAgD who are treated with BIVV009. These forward-looking statements may be accompanied by such words as "anticipate," "believe," "could," "estimate," "expect," "forecast," "intend," "may," "plan," "potential," "project," "target," "will" and other words and terms of similar meaning. You should not place undue reliance on these statements. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. Factors which could cause actual results to differ materially from Bioverativ's current expectations include: uncertainties relating to the initiation, enrollment and completion of stages of clinical trials; reliance on third parties for aspects of clinical trials; unexpected concerns may arise from data, analysis or results obtained during clinical trials or post hoc analysis of studies; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of product candidates; or Bioverativ may encounter other unexpected hurdles; and other risks and uncertainties associated with Bioverativ’s drug development and commercialization activities described in the Risk Factors section of Bioverativ’s filings with the Securities and Exchange Commission. These statements are based on Bioverativ’s current beliefs and expectations and speak only as of the date of this press release. Bioverativ does not undertake any obligation to publicly update any forward-looking statements.

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