On a quest to boost its renal diseases pipeline with two late-stage drugs, Novartis AG has announced plans to acquire precision medicines drug developer Chinook Therapeutics Inc. for up to $3.5 billion. Under terms of the deal, Novartis will acquire Seattle-based Chinook’s stock for $40 per share in cash, about $3.2 billion total, plus another $4 a share – $320 million – if certain future regulatory milestones related to Chinook’s lead product candidate, atrasentan, are met. An endothelin A receptor antagonist, artrasentan is currently in a pivotal phase III study called Align in patients with IgA nephropathy who have a high risk of kidney function decline.

Intellia touting possible ‘functional cure’ for HAE with gene-editing candidate

Promising early data continue to roll out for Intellia Therapeutics Inc.’s systemically administered CRISPR candidate, NTLA-2002, and its potential as a single-dose treatment for hereditary angioedema, a rare genetic disorder that causes inflammatory attacks on organs and tissue. Interim data for 10 patients, presented over the weekend at the European Academy of Allergy and Clinical Immunology Hybrid Congress 2023, continued to show substantial reductions in plasma kallikrein, with nine of the patients who achieved greater than 60% reductions remaining completely HAE attack-free following the 16-week primary observation period, with the longest attack-free interval of 13 months and counting. These latest results “provide the strongest evidence yet that this approach may lead to a functional cure for this disease,” said CEO John Leonard.

Newco news: Beacon launches with $121M and late-stage eye disease asset

The lead asset of Applied Genetic Technologies Corp. has been spun into Beacon Therapeutics Ltd., which launches with $120.9 million to run a phase II/III pivotal trial of AGTC-501 in X-linked retinitis pigmentosa, and to take forward two other in-licensed preclinical programs in age-related macular degeneration and cone rod dystrophy. The new company is the latest ophthalmic gene therapy play by U.K. investor Syncona Ltd., founder of Nightstar Therapeutics Ltd., which was acquired by Biogen Inc. in March 2019 for $877 million, and of Gyroscope Therapeutics Ltd., sold to Novartis AG for up to $1.5 billion in December 2021.

EY report highlights challenges, strategy for biotech sector as heady days of pandemic subside

Patent expirations, a shrinking IPO window and reduced capital availability amidst higher interest rates are just a few of the challenges faced by the biotech industry in the post-pandemic era, although the mid- to long-term industry outlook is still favorable. The 33rd edition of EY’s Beyond Borders report describes these obstacles and more faced by biotech, as well as advice for companies for traversing “a complex path forward.” Public biotech companies in the U.S. and Europe collectively recorded $215 billion in revenues in 2022, a 1% decline from 2021, according to the report.

EHA 2023: Favorable protoporphyria phase II data with bitopertin boost Disc

Shares of Disc Medicine Inc. (NASDAQ:IRON) were trading midday at $50.27, up $4.58, or 10%, after the firm rolled out positive data from an ongoing phase II open-label trial called Beacon, testing oral bitopertin, a glycine transporter 1 inhibitor, in patients with erythropoietic protoporphyria, characterized by the accumulation of protoporphyrins in red blood cells, as well as X-linked protoporphyria. The results were presented during the European Hematology Association meeting. Findings unveiled June 9 show consistent decreases in the heme precursor protoporphyrin IX, significant increases in reported sunlight tolerance, and improvements in measures of patient quality of life.

US drug negotiations face second court challenge

It didn’t take long for the filing of a second constitutional challenge to the U.S. Inflation Reduction Act’s drug negotiations. In the wake of a similar suit by Merck & Co. Inc., the Chamber of Commerce filed a complaint June 9 in the U.S. District Court for the Southern District of Ohio. With Merck’s case to be heard in the federal court for the District of Columbia, the Ohio suit could create a fast track to the U.S. Supreme Court should there be a circuit split in how the lower courts rule on the issue, especially if one issues a stay on the negotiations process scheduled to begin in September. The suit, led by the Dayton (Ohio) Area Chamber of Commerce, establishes the chamber’s standing by noting that Abbvie Inc. is a member.

EHA 2023: Gene therapy for SCD is ‘potentially’ universal in some ways, but not others

Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. As the European Hematology Association (EHA) was holding its 2023 annual congress in Frankfurt last week, Vertex announced that the FDA had accepted its rolling BLA for Exa-cel as a treatment for severe sickle cell disease (SCD) and transfusion-dependent β-thalassemia. The company’s marketing authorization application to the European Medicines Agency had been validated in January 2023. It is partly in expectation of Exa-cel’s approval that the EHA and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”

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