As Monica Bertagnolli, U.S. President Joe Biden’s choice to be the next NIH director, meets with senators to gather support for her confirmation, she’s sure to be grilled by some of them about her stance on whether the agency can march in on drug patents based on a drug’s list price. Up until now, NIH directors have said no. A change in that policy, along with nearly flatline NIH spending and other new government initiatives, could impact private investment in drug R&D by increasing the cost of research and lowering the return on investment.

AAVantgarde Bio secures $65M series A round for large gene delivery in AAV vectors

AAVantgarde Bio SrL raised €61 million (US$65 million) in series A funding to take forward two novel approaches to gene therapy that aim to overcome the packaging limits of adeno-associated virus vectors. The company aims to move its lead program, in retinitis pigmentosa associated with Usher syndrome type 1b, into the clinic later this year. A second program, in Stargardt disease, is a couple of years behind it.

Gurnet Point, Novo Holdings to acquire Paratek in transaction valued at $462M

As it nears the end of its cash runway and its lead drug Nuzyra (omadacycline) approaches a phase IIb readout that could expand the market into a rare disease indication, Paratek Pharmaceuticals Inc. agreed to be acquired by Gurnet Point Capital and Novo Holdings A/S for up to $3 per share. With 56.9 million shares outstanding at the end of the first quarter, the cash value is $122.3 million based on the price of $2.15 per share, but the transaction also includes a contingent value right (CVR) of 85 cents per share, about $48.4 million, which is payable once Nuzyra reaches U.S. sales of $320 million. Those figures added to assumed debt brings the total transaction value to $462 million. The deal is expected to close in the third quarter. The $2.15 up-front price per share is a 41% premium to Paratek’s closing stock price on May 31. Shares (NASDAQ:PRTK) rose 13% to $2.22 in early trading on June 6, following news of the merger.

ASCO 2023: Toripalimab extends OS, final phase III trial data show

When combined with chemotherapy, the PD-1 inhibitor antibody toripalimab reduced the risk of death by 37% over chemotherapy alone when used first line in patients with recurrent or metastatic nasopharyngeal carcinoma, Coherus Biosciences Inc. revealed at this year’s American Society of Clinical Oncology meeting (ASCO). The combination regimen delivered statistically significant and clinically meaningful improved overall survival (OS), with a median of 36 months vs. 33.7 months for the placebo arm, the company said, adding that median OS has not been reached in the toripalimab arm vs. chemotherapy treatment alone (p=0.0083).

Astrazeneca to offer FDA adcom an alternative to maternal RSV vaccine

Having already notched approvals in the EU and U.K., Astrazeneca plc hopes to pave the way for a third approval of nirsevimab when it makes its case June 8 before the U.S. FDA’s Antimicrobial Drugs Advisory Committee. Amid a respiratory syncytial virus (RSV) pipeline dripping with vaccines, nirsevimab, marketed as Beyfortus in Europe and partnered with Sanofi SA, is the only single-dose antibody developed to prevent RSV lower respiratory tract disease in neonates and infants born during or entering their first RSV season and in children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. If both are approved, nirsevimab could compete head-to-head with a Pfizer Inc. RSV vaccine developed to be administered in pregnancy as a way of protecting infants.

Gracell enters autoimmune space with its dual-targeting CAR T

Gracell Biotechnologies Inc. is initiating two clinical trials in China in refractory systemic lupus erythematosus (SLE) for lead candidate GC-012F, a CD19/B-cell maturation antigen (BCMA) dual-targeting CAR T. "Our lead candidate GC-012F leverages several next-generation CAR T technologies, including CD19/BCMA dual-targeting and the Fastcar next-day manufacturing,” Gracell founder and CEO William Cao told BioWorld. SLE is a new therapeutic area of interest for Gracell, as CD19-targeted CAR Ts have been reported in recent clinical research to be potentially effective in this indication.

US House committee taking aim at TRIPS waivers for COVID therapies, tests

The intellectual property waivers for American vaccines for the COVID-19 pandemic are still controversial, but the World Health Organization is still seeking a similar set of waivers for therapies and tests for COVID-19. A subcommittee of the House Judiciary Committee met June 6 to review these waivers, and subcommittee chairman Darrell Issa (R-Calif.) said he intends to continue pushing legislation that would require the U.S. president to obtain congressional approval for agreeing to any such waivers in the future, a bill that likely has the backing of manufacturers of drugs and devices virtually across the board.

Hopes for increased appropriations for FDA, NIH not necessarily lost

The Fiscal Responsibility Act (FRA) has been signed into law by U.S. President Joseph Biden, heralding a five-year span in which increases in discretionary budget spending will be limited to 1% after a flat funding picture in the coming fiscal year. While the news might seem to portend a flat budget picture for agencies such as the FDA and the NIH, a new analysis by the Alliance for a Stronger FDA indicates that congressional intent might at least slightly overcome the limits imposed by the FRA.

The BioWorld Insider Podcast: Preventing opioid overdoses with a smart patch

Free, no-prescription-required access to naloxone has reduced deaths from opioid overdoses by 11% in some communities, but they only work if someone with the reversal agent is available when a person overdoses. Resilient Lifescience Inc. hopes to save those lives, too. The company is developing a wearable patch that monitors vital signs and automatically administers naloxone when needed. In this edition of the BioWorld Insider podcast, Brad Holden, CEO of Resilient Lifescience, explained how the device works for overdoses as well as other potential applications for the new technology.

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