Fulcrum Therapeutics drug pociredir posted early clinical trial results showing signs of treating sickle cell disease with no reports of serious complications. But the biotech is discontinuing this program due to FDA concerns that drugs in this class of medicines raise the risk of cancer.
Pfizer’s inclacumab did not beat a placebo in a Phase 3 test in sickle cell disease. The trial failure comes nearly a year after Pfizer voluntarily withdrew Oxbryta from the market after tests showed higher rates of complications and deaths for that sickle cell disease drug.
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At a moment when medication therapy for sickle cell disease (SCD) leaves too many behind and widespread access to curative gene therapies still lies in the distant future, automated red blood cell exchange is experiencing renewed interest. To improve patients’ lives, a broad commitment is needed to expand access to transfusion therapies.
A multiple myeloma cell therapy has encouraging results analysts say could make it best in its class and a gene-editing therapy is showing early promise in sickle cell disease. Here’s a recap of those developments and others from the annual meeting of the American Society of Clinical Oncology.
The death wasn’t due to Beam Therapeutics’ sickle cell disease therapy, BEAM-101, but was instead attributed to the preconditioning treatment needed before the genetic medicine is infused. A different Beam program in preclinical development could avoid the need for toxic preconditioning; data from both programs will be presented during the upcoming American Society of Hematology annual meeting.
Editas Medicine says its ex vivo gene-editing therapy for hemoglobinopathies has best-in-class potential, but the company plans to out-license or partner that program. Editas is shifting focusing to in vivo gene-editing therapies.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.
Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.
Sickle cell disease is among the Beam Therapeutics programs spared, and here the company needs to show differentiation from genetic medicines under FDA review from Vertex Pharmaceuticals and Bluebird Bio. Meanwhile, Beam aims to find partners for its research in allogeneic cancer cell therapies and hepatitis B.
HPC International launched Pinpoint, an app that helps teens manage their sickle cell disease. It allows them to learn about the disease, track their pain and communicate with their physicians.
Fulcrum Therapeutics offered few details about the clinical hold the FDA placed on its Phase 1b test of its sickle cell disease drug. Graphite Bio, Intellia Therapeutics, and Sangamo Therapeutics also encountered setbacks to their sickle cell programs.
Graphite Bio voluntarily paused a Phase 1/2 test of its gene-edited therapy for sickle cell disease after the first patient in the study developed a serious blood complication. The setback will also delay plans to reach the clinic with another genetic medicine in its pipeline.
Novo Nordisk announced that it will buy Forma Therapeutics, a clinical-stage biotech focused on rare hematologic diseases, in an deal valued at $1.1 billion. The deal is the second billion-dollar sickle cell disease deal the pharmaceutical industry has seen in the last 30 days — Pfizer forked over $5.4 billion in August to buy Global Blood Therapeutics.
Pfizer’s own R&D efforts in sickle cell disease have fallen short, so the pharmaceutical giant is turning to M&A to gain a presence in the blood disorder. The acquisition of Global Blood therapeutics brings the commercialized drug Oxbryta plus a pipeline of other therapeutic candidates.
Novartis is paying Precision Bio $75 million up front to begin a partnership aiming to develop in vivo gene-editing therapies for serious genetic blood disorders, such as sickle cell disease. One of the goals of the alliance is to make genetic medicines more globally accessible.