Nearly two years after Novartis AG signed a $1.7 billion deal for options to Voyager Therapeutics Inc.’s adeno-associated virus capsids to develop central nervous system gene therapies, the company secured rights to develop gene therapies for Huntington’s disease and spinal muscular atrophy in a licensing agreement potentially worth $1.3 billion. For Voyager, the deal translates to $100 million in up-front money, including $20 million in newly issued equity, plus up to $1.2 billion in preclinical, development, regulatory and sales milestones, as well as tiered royalties earned through sales of products that come out of Voyager’s technology. The funds push out the company’s runway to mid-2026. Investors liked the deal, with shares (NASDAQ:VYGR) soaring 23% to $10.36 in early trading on Jan. 2.

Allorion joins a $540M lung cancer deal with Astrazeneca 

Allorion Therapeutics Inc. could receive $40 million in up-front and near-term payments from Astrazeneca plc in the companies’ exclusive option and global license deal. The two plan to develop and commercialize an L858R mutated allosteric inhibitor for treating epidermal growth factor receptor (EGFR) inhibitor-mutant non-small-cell lung cancer (NSCLC). Astrazeneca received the option to license the inhibitor, while Allorion is eligible for development and milestones payments of more than $500 million plus tiered royalties on worldwide net sales. Astrazeneca’s EGFR tyrosine kinase inhibitor Tagrisso (osimertinib) received the U.S. FDA’s accelerated approval for treating metastatic EGFR T790M mutation-positive NSCLC in late 2025. Privately held Allorion, which launched in 2020, dosed the first patient in its phase I/II study of ARTS-021, a CDK2 inhibitor for treating advanced hepatocellular carcinoma, in October.

Phase Ib/II encephalopathies data skyrocket Longboard stock  

Shares of Longboard Pharmaceuticals Inc. (NASDAQ:LBPH) soared 174%, or $10.52, to trade midday at $16.55 on data from the Pacific study, a phase Ib/IIa trial with bexicaserin (LP-352) in subjects with developmental and epileptic encephalopathies (DEEs). The firm said bexicaserin achieved a median seizure reduction of 53.3% in countable motor seizures compared to 20.8% in the placebo group across the DEE population. Specifically, a median drop of 72.1% turned up in Dravet syndrome, 48.1% in Lennox-Gastaut syndrome and 61.2% in other DEEs.

Celltrion to sell off ex-Takeda ‘Primary Care’ unit

Celltrion Inc. said on Jan. 2 that it reached an agreement with Asian health care investment firm CBC Group to partially sell off its Primary Care unit by March 2024 for ₩209.9 billion (US$160.7 million). The sale to CBC Group will include rights to the unit’s prescription drug products (domestically referred to as ethical drugs, or ETCs) in the Asia-Pacific region, excluding South Korea. Celltrion stock (KOSPI:068270) on the Korea Exchange soared on the news, climbing nearly 15%, or ₩30,000 (US$22.91), to close at ₩231,500 on Jan. 2.

US lawmakers: Expansion of COVID-19 IP waiver unnecessary

Six months out from the World Health Organization ending the global health emergency brought on by the pandemic, there’s no need for the World Trade Organization to expand a five-year intellectual property (IP) waiver for vaccines to COVID-19-related drugs, devices and diagnostics. So says a group of nearly 20 U.S. lawmakers in a recent letter to the U.S. Trade Representative. “The world has emerged from the depths of the pandemic, in large part a direct result of U.S. leadership,” the members of Congress said in the letter. “We should not do anything that would undermine the protections that keep U.S. research at the forefront of innovation.”

BioWorld highlights the top biopharma trends of 2023

As industry gears up for the annual J.P. Morgan Healthcare conference to kick off 2024, take a moment to review the successes and challenges that defined 2023. It was a year when biopharma faced a tough capital market, as it settled into the new post-pandemic normal, and a year when drug pricing debates went global. But the industry continued to innovate, as always, celebrating advances in gene editing and introducing the world’s first two RSV vaccines. And for companies working on GLP-1 drugs or antibody-drug conjugates, 2023 was a very, very good year.

Holiday notice

BioWorld's offices were closed Monday, Jan. 1. No issue was published.

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Aditxt, Akari, Alfasigma, Alimera, Angle, Apollo, Betterlife, Biora, Eisai, Galapagos, Graviton, Hemostemix, Immunitybio, Mannkind, Medilink, Newron, Nkgen, Odyssey Health, Oragenics, Replace, Roche, Sareum, Synimmune, Tome, Veraxa, Verona, Vicore, Wintermute, Xortx