Today, a brief rundown of news involving Prime Medicine and Saol Therapeutics, as well as updates from Revolution Medicines and Cyllene Therapeutics that you may have missed.
Prime Medicine has won a legal dispute with Beam Therapeutics surrounding rival therapies the two companies are developing for the rare disease alpha-1 antitrypsin deficiency, or AATD. Beam and Prime both sprang from the labs of gene editing specialist David Liu and, in 2019, the two companies formed a broad collaboration that, among other things, appeared to potentially forfeit Prime's rights to develop an AATD treatment. Since then, though, Prime has brought a potential therapy forward, leading Beam to claim the deal was breached and Prime to counter that the treatment was within its rights to develop. An arbitration panel ruled Wednesday in favor of Prime, meaning the company won't owe Beam any monetary damages. Beam's AATD treatment, built on "base editing" technology, is in advanced clinical development. Prime's "prime editing" rival is in preclinical testing, and could produce initial human data next year. In an emailed statement, Beam said it “respectfully disagree[s] with aspects of the ruling” but is “pleased” that the decision doesn’t affect broader, exclusive rights it has to use certain prime editing tools.
Privately held Saol Therapeutics is the latest drugmaker to announce progress resuscitating a medicine the Food and Drug Administration previously rejected. Saol on Tuesday said it resubmitted an application for SL1009, a potential treatment for the rare disease pyruvate dehydrogenase complex deficiency that the FDA turned back last August. Upon rejecting SL1009, U.S. regulators asked Saol for additional evidence to support approval. Saol provided more analyses of existing data and, after an FDA meeting in March, was advised to proceed with a new application instead of running another trial. Saol joins others, including Replimune and UniQure, that have seen their fortunes change following recent FDA meetings. Saol is "encouraged by the FDA’s demonstrated willingness to apply regulatory flexibility," CEO Dave Penake said in a statement.
European drug regulators are speeding the review of a drug that's been heralded as a breakthrough against pancreatic cancer. In a Tuesday statement, the European Medicines Agency said it's started a "phased review" of Revolution Medicines' daraxonrasib, as data becomes available and ahead of a full approval submission. According to the EMA, daraxonrasib has been recognized as a "high priority" and its expedited evaluation will be used as an "example" for expected EU legislation that could strengthen the use of phased reviews. Daraxonrasib nearly doubled survival compared to chemotherapy in a Phase 3 study earlier this year.
Cyllene Therapeutics has raised $33 million in Series C funding to support a gene therapy it’s developing for a kind of bladder malfunction associated with neurological damage. Cyllene’s gene therapies use a non-replicating version of an HSV-1 virus to deliver therapeutic DNA into cells. Its top treatment, EG110A, is designed to effectively subdue the uncontrollable spasms and resulting incontinence in people with “neurogenic detrusor overactivity.” Cyellene plans to start a late-stage trial next year.
