Bristol Myers Squibb Co. is paying $330 per share to take over Karuna Therapeutics Inc. in a deal valued at $14 billion, a move that brings aboard Karxt (xanomeline-trospium), a dual M1/M4 muscarinic acetylcholine receptor agonist under review by the U.S. FDA for schizophrenia in adults. Shares of Boston-based Karuna (NASDAQ:KRTX) were trading at $316.10, up $100.91, or 46%, while BMS’ stock (NYSE:BMY) was selling for $52.74, up $1.48. Karxt also holds potential as an adjunctive approach and first therapy for Alzheimer’s disease psychosis, with promise in other neuropsychiatric and neurodegenerative Indications.

Ionis’ Wainua approved in US for rare disease hATTR-PN

A new self-injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) will be available in January 2024 now that the U.S. FDA has approved Ionis Pharmaceuticals Inc.’s Wainua (eplontersen), a ligand-conjugated antisense oligonucleotide. Wainua was granted orphan drug designation by the FDA in January 2022. It is partnered with Astrazeneca plc through a 2021 deal worth $3.6 billion in which Ionis retained co-commercialization rights in the U.S. The partners are seeking approval in Europe, where Wainua also has orphan drug designation, and other areas of the world. Hereditary ATTR-PN leads to peripheral nerve damage with motor disability, such as the inability to walk, within five years of diagnosis. There are about 40,000 patients worldwide.

Allovir walloped by three phase III quits for futility with posoleucel

Shares of Allovir Inc. (NASDAQ:ALVR) were trading at 79 cents, down $1.54, or 66%, after the firm provided an update on posoleucel, an off-the-shelf, multi-virus-specific T-cell therapy. The drug is designed to take aim at six viral pathogens in immunocompromised people: adenovirus (AdV), BK virus, cytomegalovirus, Epstein-Barr virus, human herpesvirus-6, and JC virus. Waltham, Mass.-based Allovir will discontinue its three global phase III posoleucel studies for prevention of clinically significant infections or diseases by multiple viruses, treatment of virus-associated hemorrhagic cystitis, and treatment of AdV following allogeneic hematopoietic cell transplant. The company made the decision after the pre-planned analyses by three independent data safety monitoring boards (DSMBs), each of which recommended stopping its respective trial for futility. No safety concerns were raised by any of the DSMBs.

Antibody-drug tusamitamab ravtansine fails in phase III; Sanofi bows out

As its pharma peers continue to place big bets on antibody-drug conjugates (ADCs), Sanofi SA is ditching the only advertised ADC program in its pipeline, after it failed an independent interim analysis. The phase III failure of tusamitamab ravtansine not only puts the French pharma behind its competitors in one of the fastest growing segments of cancer therapy, it also leaves the company’s overall oncology development pipeline looking very thin.

Starpharma’s dendrimer platform meets metastatic cancer endpoints in phase II

Starpharma Holdings Ltd.’s DEP docetaxel phase II trial met its primary endpoints, demonstrating antitumor activity in multiple advanced, metastatic cancers, including pancreatic, gastro-esophageal, non-small-cell lung cancer and cholangiocarcinoma. The results come on the heels of positive phase II results for DEP-irinotecan, a dendrimer enhanced product (DEP) of the widely used drug irinotecan, marketed as Camptosar by Pfizer Inc.

Top Trends of 2023: Humira biosimilar launches top 2023 news for the follow-ons

Biosimilars grabbed a lot of headlines in 2023, thanks to the biggest U.S. biosimilar launch to date targeting Abbvie Inc.’s mega-blockbuster Humira (adalimumab). Eight biosimilars referencing the immunology drug entered the U.S. market under licensing agreements with Abbvie. Amgen Inc.’s Amjevita led the pack with a five-month headstart in January.

Top Trends of 2023: Biopharma financings settle into post-pandemic new normal

The biopharma industry is still adjusting from the heady days of the COVID-19 pandemic, and nowhere is this so apparent as in financings. The value of financings has returned to what could be called a new normal, down from the surge of capital seen in 2020-2021. However, there are signs that the trend is heading back upward at a steady pace. Already, 2023 saw a higher total financing value than 2022, as well as more total transactions.

Top Trends of 2023: First chimeric monkey with large embryonic stem cell contribution born

In November, investigators at the Chinese Academy of Sciences reported generating a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it was not the first live birth of a chimeric primate. But it was the first such chimera with contributions from an embryonic stem cell, and that stem cell contributed a far higher proportion of cells in the newborn than had been achieved in previous attempts at creating chimeras.

OIG: US NIH audit oversight puts federal funds at risk

If two recent reports about the NIH’s oversight of its grant programs were grades, the U.S. agency would have received failing marks – again. For fiscal years 2019 and 2020, foreign NIH grant recipients should have submitted a total of 109 annual audit reports. Only 28 were submitted, according to an audit by the Health and Human Services Office of Inspector General (OIG). The OIG found similar shortcomings in the NIH’s oversight of single audit requirements for extramural grant recipients, which include universities, states and localities, and for-profit Small Business Innovation Research award recipients.

FDA nods remain up from last year, yet lag behind 2017-2021

In November, the U.S. FDA approved a total of 12 drugs, a decrease from 27 the previous month that marked the highest number of approvals since June 2020, as reported by BioWorld. From January to November, FDA approvals reached 167, an increase of 21.9% from 137 drugs approved during the same time period the previous year. Approvals of new molecular entities (NMEs) are at 34 in 2023, up from 30 in the corresponding period last year. However, both total approvals and NME approvals remain down from 2017-2021.

Holiday notice

BioWorld's offices will be closed in observance of Christmas. No issues will be published Monday, Dec. 25 or Tuesday, Dec. 26.

BioWorld Insider Podcast: Six biopharma executives consider a tough 2023 and are hopeful for a better 2024

One aspect of 2023 our group of executives completely agreed on: the past year was tough financially. And they all foresee a more vibrant year ahead for the market. Giving all of them hope were technological breakthroughs such as artificial intelligence, game changing weight loss drugs, the surging fascination with ADCs and hope for lower interest rates. But uncertainty looms about the upcoming general election in the U.S. and ground-shifting gene therapies. In a preview of the annual Biotech Showcase conference, an investor conference for private and micro- to mid-cap biotech companies Jan. 8-10 in San Francisco, BioWorld spoke with Dave Bearss, CEO of Halia Therapeutics Inc., Vimal Mehta, CEO of Bioxcel Therapeutics Inc., Paul Lammers, CEO at Triumvira Immunologics Inc., Chris Pirie, COO of HDT Bio Corp., Thijs Spoor, CEO of Perspective Therapeutics Inc., and Shelley Hartman, CEO of Aegle Therapeutics Corp. They offered insights brought about by years of hard-won experience.

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