NEW YORK – Lantheus Holdings this week said the US Food and Drug Administration granted fast track designation to its radiopharmaceutical 177Lu-PNT2002 for metastatic castration resistant prostate cancer (mCRPC). Lantheus is advancing the product in partnership with POINT Biopharma in a Phase III trial involving patients with PSMA-expressing mCRPC who have progressed on androgen receptor pathway inhibitor therapy and refuse, or are not eligible for, chemotherapy. Lantheus has worldwide commercialization rights to 177Lu-PNT2002, except for certain Asian territories, under a licensing agreement signed with POINT in December 2022. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval.
Avidity Biosciences this week said it's been granted fast track designation from the US Food and Drug Administration for AOC 1044, a therapy designed to treat Duchenne muscular dystrophy in patients with certain mutations amenable to exon 44 skipping. AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers to patients' skeletal muscle and heart tissue, which skips exon 44 of the dystrophin gene and enables production of functional dystrophin protein. AOC 1044 is currently in a Phase I/II trial.
Health-tech company PicnicHealth this week said it plans to expand a data-sharing partnership it originally entered with Roche in 2018, under which the pharmaceutical company and its subsidiary Genentech now will be able to access PicnicHealth's newest datasets in Alzheimer's disease-related dementias and myasthenia gravis. The datasets from PicnicHealth comprise anonymized information on patient- and observer-reported outcomes as well as electronic health record data, including results from molecular and diagnostic lab tests and genetic testing.
Shanghai-based gene therapy developer Belief BioMed this week announced that it has dosed all subjects in a registrational and Phase III clinical trial for BBM-H901, an adeno-associated virus (AAV) gene therapy designed as a prophylactic treatment for bleeding in adults with hemophilia B. It's the first AAV gene therapy for hemophilia B with investigational new drug approval for registrational clinical study in China, the company said.
The UK Medicines and Healthcare Products Regulatory Agency has granted Wakefield, Massachusetts-based Myrtelle innovative licensing and access pathway designation for its drug candidate for Canavan disease, the company said this week. The drug, rAAV-Olig001-ASPA, is a recombinant adeno-associated virus (rAAV) that targets brain cells that produce myelin and is intended to restore function in the ASPA gene. Myrtelle, a gene therapy developer focused on neurodegenerative diseases, entered into an exclusive worldwide licensing agreement with Pfizer in 2021 to develop and commercialize the therapy, which is now its lead product candidate.
Private equity firm TA Associates plans to make a strategic growth investment in Diatech Pharmacogenetics, the Italian pharmacogenetics and cancer precision medicine diagnostics company announced this week. The investment, expected to close in Q2, will support Diatech's efforts to expand into markets internationally. Financial terms were not disclosed, but as part of the deal, Diatech leaders will retain majority ownership of the company while minority investor Alto Partners will exit its stake.
Bristol Myers Squibb said this week that it will add in-house viral vector production to its cell therapy manufacturing network via a US-based manufacturing facility in Libertyville, Illinois, pursuant to an agreement with Novartis. The facility and its operations will produce viral vector for Bristol Myers Squibb's CAR T-cell therapies. Bristol Myers Squibb will take over the new US facility in 2023, upon fulfillment of closing conditions.
Malvern, Pennsylvania-based biotechnology company Ocugen this week said the US Food and Drug Administration had granted orphan drug designation to OCU410ST (AAV5-hRORA), a gene therapy designed to treat retinopathies associated with the ABCA4 gene. Ocugen plans to submit an investigational new drug application in Q2 to begin a Phase I/II trial for the drug.
The US Food and Drug Administration has granted orphan drug designation to EDIT-301, gene-editing company Editas Medicine's investigational therapy for sickle cell disease, the firm said this week. EDIT-301 contains patient-derived CD34+ hematopoietic stem and progenitor cells with edited gamma globin gene (HBG1 and HBG2) promoters and is designed to create a sustained increase in fetal hemoglobin production. The Cambridge, Massachusetts-based company plans to dose 20 patients in a Phase I/II trail assessing EDIT-301 for severe sickle cell disease by year-end.
Ideaya Biosciences this week closed its underwritten public offering of 8,858,121 shares of its common stock at $18.50 per share and pre-funded warrants to purchase 2,020,270 shares of common stock at $18.49 per share. The firm also said the underwriters fully exercised their option to purchase an additional 1,418,920 shares of common stock. The gross proceeds from the offering were approximately $201 million.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.