About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle (LNP) delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies. Initially, the deal with Vertex entails $700 million, including $65 million up front and a potential $635 million in milestone payments for sickle cell disease and transfusion-dependent beta thalassemia products. But upon Vertex’s option, the size could balloon to another $365 million in milestone payments for each of 10 additional indications, bringing the potential grand total of the deal to $4.35 billion for Orna. “This is our second major pharma alliance in the last two-and-a-half years or so,” Orna Chief Scientific Officer Joe Bolen told BioWorld. In August 2022, Orna joined with Merck & Co. Inc. in a $3.7 billion deal focused on novel circular RNA protein expression technology.

Avenzo, Duality in $1B-plus pact for cancer ADC

Avenzo Therapeutics Inc. signed an exclusive license agreement with Duality Biotherapeutics Inc., whereby Avenzo will develop, manufacture and commercialize AVZO-1418/DB-1418, a potential best-in-class EGFR/HER3 bispecific antibody-drug conjugate, globally (excluding greater China). Duality is banking an up-front payment of $50 million and is eligible to collect up to about $1.15 billion in development, regulatory and commercial milestone rewards, plus tiered royalties in Avenzo’s territory. IND-enabling studies are ongoing, with plans to advance the compound into a first-in-human study this year. EGFR and HER3, the firms pointed out, are co-expressed across various solid cancers, including non-small-cell lung, breast, and head and neck.

Hoth’s phase IIa derma data in cancer patients propels its stock

Strong interim safety and efficacy data from Hoth Therapeutics Inc.’s phase IIa study of HT-001 helped boost the company’s stock (NASDAQ:HOTH) at midday as shares had risen by nearly 200%. The therapy, which inhibits recruitment and activation of immune cells, was designed to treat skin toxicities linked to EGFR inhibitors found in cancer patients. Results from the open-label portion of the study showed all patients hit the primary efficacy endpoint of improvements in skin toxicity at six weeks. About two-thirds of the participants also had lower pain and itching cores, and no treatment-related adverse effects were reported.

Novartis closing Morphosys facilities, laying off 330 workers

The curtain is coming down on one of Europe’s longest-established biopharmas, with Novartis AG announcing it is to shut Morphosys AG’s facilities, following its 2024 acquisition of the one-time antibody pioneer for $2.9 billion. The closure of sites in the U.S. and Germany by the end of 2025 will affect 330 employees. The decision was presented as being the result of a regular evaluation of the portfolio and organizational structure of the pharma company; however, the specific reason is a huge hiccup in the development of the myelofibrosis therapy pelabresib, the lead asset in the Morphosys portfolio.

China approves country’s first mesenchymal stem cell therapy

China’s health regulator gave conditional approval to Platinum Life Excellence Biotech Co. Ltd.’s amimestrocel injection (hUC-MSC PLEB-001, Ruibosheng) as the nation’s first human umbilical cord-derived mesenchymal stem cell therapy to treat steroid-refractory acute graft-vs.-host disease (SR-aGvHD) on Jan. 2, 2025. Amimestrocel is an allogenic umbilical cord-derived MSC therapy for SR-aGvHD patients ages 14 and older. Approval of the intravenous infusion was issued through the NMPA’s accelerated market registration track, which is designed for novel or urgently needed medicines, and allows its use in mainland China.

Dimerix licenses phase III kidney disease drug to Fuso for ¥10.5B

Dimerix Ltd. and Fuso Pharmaceutical Industries Ltd. signed a license agreement for the development and commercialization of Dimerix’s phase III candidate, DMX-200, for the treatment of focal segmental glomerulosclerosis (FSGS) in Japan for ¥10.5 billion (US$66.5 million) plus royalties. Under terms of the deal, Fuso is acquiring exclusive rights to develop, register and commercialize DMX-200 for FSGS in Japan. In exchange, Dimerix will receive ¥300 million within 40 days of execution of the agreement, ¥400 million for the first development milestone of opening the first clinical site in Japan, up to ¥3 billion in further potential development milestones, and up to ¥6.8 billion in potential sales milestones, plus 15% to 20% royalties on net sales.

US FDA weighs in on timing of confirmatory trials

What does it mean for a confirmatory trial to be “underway”? That’s a question that’s been plaguing some drug sponsors since the U.S. Congress, in 2023, gave the FDA the authority to require, as a condition of accelerated approval, that a confirmatory trial be underway at the time of the approval. To answer the question, the FDA is releasing a draft guidance discussing policies for implementing the requirement, including factors the agency intends to consider when determining whether a trial is underway.

Athira to pay $4M to resolve research misconduct allegations

Stressing the importance of integrity in taxpayer-funded biomedical research, the U.S. Department of Justice reported that Athira Pharma Inc. has agreed to pay more than $4 million to resolve False Claims Act allegations that it failed to report potential research misconduct to the NIH and the Department of Health and Human Services’ Office of Research Integrity in grant applications and progress reports. “The partnership between the scientific community and the federal government is built on trust and shared values of ethical scientific conduct,” Principal Deputy Assistant Attorney General Brian Boynton said in announcing the settlement yesterday.

The BioWorld Insider podcast: a year of interviews and insights

The BioWorld Insider podcast is a monthly dive into the business and science of drug development. Conversations with CEOs and BioWorld analysts in the past 12 months have included a wide range of chats with experts who are shaping the future. Subjects included quarterly reviews for M&As and financings, the slow but steady growth of funding and partnering for therapies to treat women, the impact of artificial intelligence and quantum computing on drug development, rare disease drug development, the costs of delays in clinical trials, and an overview of the one of the year’s biggest conferences, the annual American Society of Clinical Oncology gathering in Chicago.

2024 BioWorld Year in Review

BioWorld’s staff recaps the therapeutic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as biopharma forges ahead in 2025.

2024 BioWorld MedTech Year in Review

BioWorld MedTech’s staff recaps the med-tech and diagnostic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as med tech forges ahead in 2025.

2024 BioWorld Science Year in Review

Taking a look back at the achievements in research this year, including brain maps, pandemic breakthroughs and advances in pregnancy health.

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Acurx, Al Proteins, Alzheon, Amgen, Aro, ARS, Astellas, Astrazeneca, Bright Minds, City, Crispr, Cyprium, Eccogene, Fortress, Harrow, I-Mab, JCR, Kura Oncology, Lpoxy, Mannkind, Modalis, Naya, NMD, Nuvation Oculis, Paradox, Portage, Quoin, Rakovina, Renovaro, Rycarma, Sentynl, Sling, Star, Technoderma Medicines, Tempest, Xeno, Xortx