Eli Lilly and Co. is buying Verve Therapeutics Inc. and its gene-editing program for about $1.3 billion. Two of the one-time treatments are in the clinic. Lead candidate VERVE-102, a gene-editing treatment targeting PCSK9, is in a phase Ib study to reduce cholesterol levels. Verve and Lilly began their partnership in 2023, with Verve receiving $60 million in an up-front payment plus an equity investment and eligibility for up to $465 million in R&D and commercial milestone payments. Verve’s investors liked the acquisition, as the stock (NASDAQ:VERV) was up 79% to $11.23 at midday.

New orphan drug, CSL’s garadacimab, cleared for hereditary angioedema

Following a complete response letter issued last October over CMC issues, CSL Behring LLC gained U.S. FDA approval June 16 of its humanized anti-factor XIIa monoclonal antibody, garadacimab (CSL-312), to prevent hereditary angioedema attacks (HAE). The U.S. approval comes on the heels of other major markets. Garadacimab won approval for HAE in January in Australia and the U.K., BioWorld previously reported. It was subsequently approved in the EU, Switzerland, Japan and the United Arab Emirates. It is marketed as Andembry in all seven places.

Alphadesign: AI creating proteins from scratch

The Alphafold machine learning system for predicting a protein’s structure from its amino acid sequence has been inverted to make it possible to design de novo proteins that fold in a particular way and bind to prespecified target proteins. The sister system, called Alphadesign, works by generating random strings of amino acids, using Alphafold to predict their structure, and then iteratively optimizing the design. A paper published today, June 17, in Molecular Systems Biology demonstrates that predicted structures have structural integrity and that the method enables accurate protein design.

Nextcure stock drops near 30% on $745M ADC deal with Simcere

Shares of Nextcure Inc. (NASDAQ:NXTC) dropped 26.27% on news of a potential $745 million partnership with Simcere Zaiming for Simcere’s cadherin-6 antibody-drug conjugate (ADC) candidate. The potential $745 million deal announced June 16 granted Nextcure worldwide rights, excluding China, Hong Kong, Macau and Taiwan, for Simcere’s SIM-0505 for solid tumors. Under the terms, Simcere is eligible to receive a total of $745 million from undisclosed up-front, development, regulatory and sales milestone payments, along with potential tiered royalties up to double digits.

Too much at stake for dismissed ACIP members to go quietly

The 17 members abruptly terminated last week from the U.S. CDC’s Advisory Committee for Immunization Practices (ACIP) are not going gently into the night. Instead, they’re raging against what could be the dying of the light. The 17 raised their collective voices in a JAMA opinion piece published yesterday to decry what’s at stake with Health and Human Services Secretary Robert Kennedy’s efforts to cleanse ACIP of what he claimed were conflicts due to financial ties to industry. So what is endangered? The health of millions of children, access to proven vaccines and the U.S. immunization policy itself, according to the former ACIP members.

BIO 2025: Bridging the gap with a ‘molecule-to-medicine expressway’

For years, the biopharma industry has spent increasing amounts of money on R&D without improving success rates, leaving many executives searching for new, more predictable drug development paths. On the second day of the Biotechnology Innovation Organization’s annual convention in Boston, Karim Budhwani, co-founder and CEO-scientist of Cerflux, moderated a panel of biopharma executives for the fourth consecutive year to discuss how companies can bridge “the valley of death” and move to a “molecule-to-medicine expressway” to improve drug discovery and development using AI and machine learning. It’s the “best panel at BIO four years in a row,” Budhwani said. “Do you know who our opening act was?” (Former U.S. President George W. Bush headlined the day with an event that was closed to media.)

Dyne sinks on endpoint change in DM1 study

Shares of Dyne Therapeutics Inc. (NASDAQ:DYN) were trading at $11.28, down $2.54, or 18%, after disclosing two pieces of news. The firm attended a type C meeting with the U.S. FDA regarding the prospects for DYNE-101 for myotonic dystrophy type 1 (DM1). At the sit-down, Dyne agreed to change the primary endpoint of the ongoing registrational expansion cohort of the phase I/II Achieve trial from the composite alternative splicing index (CASI-22) to the video hand opening test (vHOT). CASI-22 measures the correction of splicing abnormalities, while vHOT tracks the time it takes for a person to open the hand after maximal handgrip. Dyne also said the FDA has granted breakthrough therapy designation to DYNE-101, an antisense oligonucleotide for DM1, also known as Steinert’s disease.

Also in the news

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