With World's Most Costly Drugs, Uncertainty Looms Over Bluebird's Financial Future

 

• Bluebird Bio Inc 
  BLUE-3.16%+ Free Alerts
   is about to become the seller of the two most expensive drugs in the U.S. and probably the world. The price tag of $3 million is already facing backlash.
• Two FDA approvals this year might have saved the company from the financial abyss. But Bluebird will be cash-strapped for a while, the WSJ reported.
• In April, Bluebird laid off around 30% of its workforce.
• In September, the FDA approved Skysona to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
• In August, FDA approved Bluebird's Zynteglo (beti-cel), a one-time gene therapy custom-designed for beta‑thalassemia.
• Beta thalassemia and CALD afflict tiny portions of the population, limiting their economic potential.
• The report cited an analyst at RBC Capital Markets that with only an estimated 1,300 people with transfusion-dependent beta-thalassemia in the U.S., sales for the two treatments might reach just over $200 million by 2030.
• The company expects 2022 cash burn of almost $340 million. As of the second quarter, it had about $218 million in cash and equivalents.
• The company "has a clear path to financial sustainability and considerable positive momentum as we approach the anticipated BLA filing for lovo-cel for sickle cell disease in Q1 2023," the report cited a spokeswoman.
• While approval certainly would boost the company's prospects, it could still face competition from other gene therapy players like Vertex Pharmaceuticals Inc 
  VRTX+2.73%+ Free Alerts
   and CRISPR Therapeutics AG 
  CRSP-3.44%+ Free Alerts
  .

https://www.benzinga.com/general/biotech/22/10/29123370/bluebird-bio-offers-worlds-most-expensive-drugs-but-uncertainty-looms-over-its-financial-future

Kezar OKd for Autoimmune Hepatitis IND

 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced that it has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration for zetomipzomib, its first-in-class, selective immunoproteasome inhibitor, for the treatment of autoimmune hepatitis (AIH).

AIH is a rare, chronic disease in which the immune system attacks the liver and causes inflammation and tissue damage, severely impacting patients’ physical health and quality of life. Lifelong maintenance therapy is required to avoid relapse and burdensome adverse effects. If left untreated, AIH can lead to cirrhosis, liver failure and hepatocellular carcinoma. In the United States, AIH affects approximately 140,000 individuals, with incidence rates increasing. The cause of this condition remains unclear. Females are affected four times as often as males. Standard of care treatment for AIH is immunosuppressive treatment with chronic corticosteroids that can lead to additional morbidity and mortality. There is a significant need for treatment regimens that reduce or remove the need for chronic immunosuppression using corticosteroids.

The PORTOLA trial (KZR-616-208) is a randomized, double-blind, placebo-controlled Phase 2a clinical trial evaluating the safety and efficacy of zetomipzomib in patients with AIH that are insufficiently responding to standard of care or have relapsed. The target enrollment will be 24 patients, who will be randomly assigned (2:1) to receive either zetomipzomib with prednisone or placebo with prednisone for 24 weeks, with a protocol-directed steroid taper by Week 14. The primary efficacy endpoint will measure the proportion of patients who achieve a complete response measured as normalization of alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels with a successful corticosteroid taper by Week 24.

https://finance.yahoo.com/news/kezar-life-sciences-receives-fda-200100416.html

BioCardia's Heart Failure Cell Therapy Shows 100% Survival At Two Years

 

• BioCardia Inc 
  BCDA+4.12%+ Free Alerts
   announced data from its CardiAMP Cell Therapy for Heart Failure pivotal trial.  
• "The two-year outcomes for patients with heart failure receiving the investigational CardiAMP Cell Therapy in the roll-in cohort showed clinical improvement with 100% survival over two years. These outcomes surpassed our expectations in terms of patient benefit across prespecified primary and secondary endpoints," said Peter Altman, BioCardia's President & CEO.
• "While this smaller cohort is not a head-to-head comparison, the current state of the art therapies for these patients which have been successful in slowing disease, are assessed by us to have 79.9% survival after two years."
• Improvement in median functional capacity as measured by a six-minute walk distance was observed by six months (28.5 m), with a six-minute walk distance maintained through 24 months (31.0 m).
• In the study, 70% of patients reported improved or stable quality of life over 24 months.
• At 24 months, 50% of patients were improved by at least one NYHA class (n=4 at class I), 20% had unchanged NYHA class, and 30% deteriorated by one class, from class II to III.

https://www.benzinga.com/general/biotech/22/10/29121551/biocardias-heart-failure-cell-therapy-shows-100-survival-at-two-years