Global Blood Therapeutics buoyed by good news from FDA; Cigna to help cover pricey therapies
→ Global Blood Therapeutics $GBT has much to cheer about. Not only has the FDA granted its sickle cell disease (SCD) drug priority review, the agency is not holding an advisory committee to deliberate on the drug. Shares of the San Francisco-based biotech jumped more than 14% to $54.25 before the bell on Thursday. The US regulator is expected to decide on the once-daily pill voxelotor — which is designed to work by increasing hemoglobin’s affinity for oxygen — by February 26, 2020.
The field for SCD therapies is getting crowded. Months ago, Novartis‘ $NVS SCD drug, crizanlizumab, secured priority review for its ability to prevent debilitatingly painful vaso-occlusive crisis in patients. Other drugmakers, including bluebird bio $BLUE, Imara, and partners CRISPR Therapeutics $CRSP and Vertex $VRTX, are also working on their own therapies. Pfizer $PFE and GlycoMimetics $GLYC are testing their SCD drug, rivipansel, in an ongoing Phase III study. In 2017, Emmaus Life Sciences secured SCD approval from the FDA for Endari — a pharmaceutical version of L-glutamine, although the EU regulators did not back the drug.
→ Health insurer Cigna $CI, which late last year merged with Express Scripts, has found a way to cover pricey gene therapies. As part of a new offering, it will now cover Luxturna, approved for rare cases of genetic blindness, from Spark Therapeutics — a company which is in the long process of being acquired by Roche — as well as the world’s most expensive gene therapy, Novartis‘ $NVS Zolgensma, for an inherited muscle-wasting condition called spinal muscular atrophy. Consumers who are covered under the new program will have no out-of-pocket payments related to the cost of the medications, the company said on Thursday.
→ Days after unveiling plans to make a $60 million Nasdaq debut, Montreal-based Bellus Health has upsized its IPO, raising $70 million by offering about 9.9 million shares at $7.10/share. Bellus has positioned itself as David to Merck‘s Goliath in the field of chronic cough by developing drugs to block the P2X3 receptor, which is understood to play a role in hypersensitivity disorders. In its IPO filing, Bellus underscored that Merck’s late-stage drug, gefapixant, triggered taste loss or alteration in over 70% of patients who received 50 mg of the drug in its latest trial — versus 5% of patients in Bellus’s study. In July, Bellus began its Phase II trial for chronic cough, which affects 26 million Americans.
→ After their topical treatment for alopecia areata, licensed from Rigel Pharma, failed a mid-stage study back in June — tumbling their stock by 32.58% — Aclaris Therapeutics is laying off 86 emloyees and looking to partner out some programs to help right the ship. The newly completed strategic review calls for a trimming of Aclaris’ salesforce, offloading the responsibility of commercializing certain products to another company. The programs include Rhofade (oxymetazoline hydrochloride) cream, 1%; their drug candidate A-101 45% topical solution (a potential treatment for verruca vulgaris or common warts); and the drug candidates ATI-501 (oral) and ATI-502 (topical), which are investigational Janus kinase (JAK) 1/3 inhibitors for the potential treatment of alopecia.
Aclaris will continue the development of ATI-450 (investigational oral MK2 inhibitor), currently in a Phase I clinical trial for rheumatoid arthritis, and for ATI-1777. The terminations are estimated to cost between $3.0 million and $3.5 million over the next 6 months.
→ Schrödinger announced the expansion of its multi-year, multi-target drug discovery collaboration with Takeda, which comes a few months after the company received an additional $110 million backing in its first venture round. The company says that the “expansion increases the potential number of drug discovery programs that can be initiated under the collaboration and increases the cumulative value of potential future milestone payments that Schrödinger is eligible to receive per program.”