Kalvista Pharmaceuticals Inc. is promising to “change the paradigm” in the treatment of hereditary angioedema (HAE), after announcing positive phase III data for its oral on-demand kallikrein inhibitor sebetralstat. Rather than waiting four to five hours to self-administer therapy, as is the case for approved injectable on-demand therapies, there was a median time to dosing of 10 minutes after the start of an attack in the 136-patient placebo-controlled trial. The data are “the best-case scenario,” in the view of analyst Joseph Schwartz at Leerink Partners. Kalvista is now preparing to file an NDA with U.S. FDA in the first half of 2024, and in Europe and Japan later in the year.

Roche bails on camonsertib, Repare regains full rights

Weeks after Roche Holding AG paid a $40 million milestone payment to partner Repare Therapeutics Inc. when the first cancer patient in the phase II Tapistry trial was dosed with camonsertib, the Basel, Switzerland-based pharma backed out of the 20-month-old deal. The decision restores to Repare full global development and commercialization rights to camonsertib (RP-3500), an oral, small-molecule inhibitor of ATR (ataxia-telangiectasia and rad3-related protein kinase). The $1.33 billion oncology deal, signed by the companies in June 2022, officially ends May 7, 2024, a move resulting from “a review of Roche’s pipeline and evolving external factors,” according to Repare. This is the second major collaboration terminated by Roche in the last two weeks. On Jan. 29, it dissolved its October 2022 deal with Hookipa Pharma Inc. for the HB-700 program to treat KRAS mutated cancers.

$170M series D fuels Bioage push in obesity

Bioage Labs Inc. scored a $170 million series D financing to boost phase II trials with azelaprag, an apelin receptor agonist, in combination with Zepbound (tirzepatide), the glucose-dependent insulinotropic polypeptide receptor and glucagon-like peptide-1 (GLP-1) receptor agonist from Bioage partner Eli Lilly and Co. Azelaprag is designed to improve metabolic and muscle function and could work as an oral drug to increase weight loss and improve body composition in patients on GLP-1/incretin therapy, Richmond, Calif.-based Bioage said.

Biogen’s ataxia drug, Crispr’s sickle cell therapy cleared in EU

The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).

Three strikes for Otsuka’s AVP-786 as it fails in AD-related agitation

Otsuka Pharmaceutical Co. Ltd.’s AVP-786 missed the primary endpoint for a third time in a phase III trial for agitation associated with dementia due to Alzheimer’s disease. Two previous phase III trials also failed to show statistical significance for AVP-786. The trial was ongoing before the U.S. FDA cleared Otsuka’s Rexulti (brexpiprazole) in May 2023, as the first approved drug for agitation in Alzheimer’s dementia. Regardless, a statistically significant difference was not achieved on the primary efficacy endpoint, mean change from baseline to week 12 in the Cohen-Mansfield Agitation Inventory total score between AVP-786 and placebo. In addition, a treatment-emergent adverse event, a fall, appeared with an incidence rate of more than 5% in patients treated with AVP-786 and greater than placebo.

Lilly and Vertex lead BioWorld Biopharmaceutical Index gainers in 2023

Concluding 2023 on a strong note, the BioWorld Biopharmaceutical Index (BBI) ended the year up 11.62%, surpassing the end of November increase of 3.37%. BBI kicked off the new year with a solid 4.77% increase by January’s end. Leading the pack in 2023 was Eli Lilly and Co. (NYSE:LLY), posting a 59% gain, followed by Vertex Pharmaceuticals Inc. (NASDAQ:VRTX), climbing 41% over the year. Pfizer Inc. experienced the largest downturn, with its shares (NYSE:PFE) plummeting 44% in 2023.

HHS scores first win in challenges to US price negotiations

One down, eight to go. That’s the scorecard for the constitutional challenges to mandatory Medicare drug price negotiations now that a U.S. federal court has dismissed a suit filed by the Pharmaceutical Research and Manufacturers of America, the National Infusion Center Association and the Global Colon Cancer Association. Judge David Ezra, of the U.S. District Court for the Western District of Texas, yesterday granted Health and Human Services (HHS) Secretary Xavier Becerra’s motion to dismiss the challenge, finding that the court lacked subject matter jurisdiction and that the Texas-based court was an improper venue for the suit.

US PTO guidelines for AI suggest amplified need for documentation

The U.S. Patent and Trademark Office has released a draft version of patent examiner guidelines to address the increasing use of artificial intelligence (AI) in the inventive process, reflecting the standing U.S. position that AI cannot be an inventor. However, these new guidelines reinforce the notion that inventors should meticulously document the inventive process if they are to avoid problems that could arise in connection with the role of the AI in that process, a failure of which could invalidate one or more claims in the patent application and significantly damage or even destroy the value of the patent.

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