NEW YORK – The US Food and Drug Administration this week granted fast-track designation to Repare Therapeutics' PKMYT1 inhibitor lunresertib in combination with its ATR inhibitor camonsertib for the treatment of CCNE1-amplified or FBXW7- or PPP2R1A-mutated platinum-resistant ovarian cancer. Repare is currently studying the lunresertib-camonsertib combination in a cohort of the Phase I MYTHIC trial. The firm expects to report data from the combination in this cohort in the fourth quarter of 2024. Earlier this year, Repare regained rights to camonsertib after Roche ended its collaboration agreement for the drug. With fast-track designation, a sponsor can meet with the FDA more frequently to get advice on developing fast track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
Delfi Diagnostics this week said that the Merck Global Health Innovation Fund, the venture capital arm of Merck & Co., has invested in Delfi to help the company accelerate and expand the development of its cancer detection tests. Delfi's fragmentomics technology leverages AI to whole-genome sequencing data to assess a patient's cell-free DNA patterns and characteristics against individuals with and without cancer. Delfi declined to disclose the amount of the investment.
The US Food and Drug Administration has granted Cambridge, Massachusetts-based Be Biopharma orphan drug designation for BE-101, an autologous engineered B-cell therapy for hemophilia B, the company said this week. Be Bio said it is on track to begin a Phase I/II trial of the investigational therapy, known as the BeCoMe-9 study, in adults with severe or moderately severe hemophilia B in the second half of the year.
New York-based Myrtelle this week said one of its gene therapy candidates, rAAV-Olig001-ASPA, has been selected for the US Food and Drug Administration's Support for clinical Trials Advancing Rare disease Therapeutics, or START for short, a pilot program the agency launched last year. The gene therapy is being developed as a potential treatment for Canavan disease, a genetic brain disease caused by mutations in the ASPA gene that result in impaired brain development.
Cambridge, Massachusetts-based Moderna this week also announced that mRNA-3705, its investigational mRNA therapeutic for methylmalonic acidemia caused by a deficiency in the mitochondrial enzyme MUT, was selected for the START pilot program. The drug candidate comprises an mRNA that encodes for MUT delivered by a lipid nanoparticle.
Pierre Fabre Laboratories said this week that it has filed an investigational new drug application with the US Food and Drug Administration for a Phase I/II trial of its investigational agent PFL-002/VERT-002 in MET-altered solid tumors. If the FDA clears the IND, Pierre Fabre will begin studying the monoclonal antibody, which was developed by Vertical Bio, in patients with solid tumors, including non-small cell lung cancer, harboring cMET alterations. Pierre Fabre hopes to enroll the first patient in the first-in-human trial by the end of 2024.
OpGen said this week that the Nasdaq has granted the company its request for continued listing on the exchange while OpGen tries to file its first quarter Form 10-Q with the US Securities and Exchange Commission. The Nasdaq had told the firm last week it did not meet a listing requirement because it had not filed its SEC form in a timely manner. The Nasdaq has given OpGen until July 8 to file its Form 10-Q.
Liquid biopsy firm Angle reported this week preliminary financial results that indicate its revenues rose 120 percent for the full fiscal year 2023 to £2.2 million ($2.8 million) compared to £1.0 million in the prior year. The firm said that it expects to record a loss of £20.1 million, or £.0773 per share, compared to a loss of £21.7 million, or £.0879 per share, a year earlier. Angle said that it ended the year with £16.2 million in cash and cash equivalents. The firm also said that it anticipates first half 2024 revenues in the range of £1.0 million to £1.3 million and that the firm will deliver strong growth during the year.
GeneDx said this week that it has started a patient access program, developed in partnership with biopharma companies, to increase access to exome sequencing for pediatric epilepsy patients. Patients must meet a number of criteria to be eligible for the program, which is available to select ordering providers immediately and will be expanded in July.
Becton Dickinson announced this week that it will acquire the critical care product group Edwards Lifesciences for $4.2 billion in cash. The critical care business's portfolio includes AI-enabled clinical decision tools and hemodynamic patient monitoring using AI algorithms, which BD said will integrate with its medication management and smart connected care solutions. Based in Irvine, California, the critical care business has more than 4,500 employees and generated $900 million in revenues last year. BD expects to fund the all-cash transaction with approximately $1 billion in cash and $3.2 billion in new debt and said the transaction will be immediately accretive to revenue growth, adjusted gross margin, adjusted operating margin, and adjusted EPS. The transaction is expected to close by the end of 2024, subject to regulatory reviews and closing conditions, and the business will then operate as a separate unit within BD's medical segment.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.