Shares of Adverum Biotechnologies Inc. (NASDAQ:ADVM) sank 18% to trade midday at $2.29, down 52 cents, after the firm unveiled preliminary safety and efficacy data from the ongoing Luna phase II trial with gene therapy ixoberogene soroparvovec (ixo-vec) in patients with wet age-related macular degeneration (AMD). Redwood City, Calif.-based Adverum said both dose levels of ixo-vec turned up potential best-in-class activity in challenging AMD patients, including treatment burden reduction and maintenance of visual acuity as well as good results with regard to anatomic endpoints. Preliminary safety data support a favorable benefit-risk profile, too, the firm said.

Biontech and Autolus collaborate to advance their CAR T programs

For $50 million cash, Biontech SE bought the rights to use Autolus Therapeutics plc’s manufacturing and commercial infrastructure in the U.K. so it can advance its CAR T-cell BNT-211 program in the clinic. Biontech has at least 10 registrational studies in its pipeline for 2023, which includes BNT-211 in relapsed or refractory germ cell tumors. Biontech will pitch in on the launch and expansion of development program of Autolus’ lead cell therapy candidate CD19 CAR T therapy, obecabtagene autoleucel (obe-cel), and will receive a royalty on net sales. Obe-cel has a Nov. 16 PDUFA from the U.S. FDA for treating relapsed/refractory adult B-cell acute lymphoblastic leukemia. Biontech, in a private placement, also will buy $200 million of Autolus’ American depositary shares. Autolus also priced an underwritten offering in the U.S. of 58.3 million American depositary shares at $6 each for proceeds of $350 million. The company plans to use the net proceeds of the offering plus the $250 million from Biontech, to advance the obe-cel program and fund its manufacturing.

Kyowa pays $100M up front for Bridgebio’s infigratinib in Japan

Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal. Announced on Feb. 7, Kyowa will pay $100 million up front and royalties up to the high-20s percent on infigratinib sales in Japan, along with potential milestone payments. In turn, QED Therapeutics Inc., a Bridgebio affiliate, grants Tokyo-based Kyowa exclusive licensing rights to develop and commercialize infigratinib for achondroplasia, hypochondroplasia and other skeletal dysplasias in Japan.

Regenxbio’s Hunter syndrome gene therapy hits goals in pivotal trial

Regenxbio Inc. plans to file a BLA this year seeking accelerated approval for gene therapy candidate RGX-121 to treat young children with mucopolysaccharidosis type II, also known as Hunter syndrome, based on positive data from the phase I/I/III Campsite trial, which not only hit the biomarker endpoint but also indicated potential systemic benefits.

US Senate drug pricing hearing more than ‘CEO-whack-a-mole’

Today’s U.S. drug pricing hearing before the Senate Health, Education, Labor and Pensions Committee started out as a spectacle in which Chair Bernie Sanders (I-Vt.) rehashed his usual talking points as he preached at the CEOs of Bristol Myers Squibb Co., Johnson & Johnson and Merck & Co. Inc. over how their companies charge so much more for their drugs in the U.S. than in other countries. But despite the show-trial aspects, or what Ranking Member Bill Cassidy (R-La.) called Sanders’ “CEO-whack-a-mole” agenda, some facts came through that could lead to pricing reforms if Congress has the bipartisan will to do so.

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60 Degrees, Alector, Biocomposites, Biocorrx, Briacell, Bristol Myers Squibb, Camino, Cantabio, Cassava, Coeptis, Denali, Domain, Eli Lilly, Foghorn, Genprex, Hemostemix, Hutchmed, Immix, Inhibikase, Innovent, Invizius, Krystal, Kyverna, Mineralys, Myrobalan, Neurona, Prokarium, Qsam, Regenxbio, Sana, Telix, Tenax, Tenaya, Theriva, Zymeworks