Neurocrine Biosciences Inc. unveiled positive top-line data from the phase III study called Cahtalyst testing the efficacy, safety and tolerability of crinecerfont in adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The trial met its primary endpoint at week 24, demonstrating that treatment with crinecerfont resulted in a statistically significant percent reduction in daily glucocorticoid dose vs. placebo while maintaining androgen control. CAH is caused by genetic defects in the proteins and enzymes involved in cortisol biosynthesis.

Acelyrin’s izokibep misses primary HS goal on patient dropouts and perplexing placebo hikes

Acelyrin Inc.’s shares tumbled after its interleukin-17A inhibitor, izokibep, failed to reach statistical significance in part B of a phase IIb/III trial in moderate to severe hidradenitis suppurativa (HS), but the company remains undeterred with its plans to advance the drug for the inflammatory skin condition. The primary endpoint, Hidradenitis Suppurativa Clinical Response 75 (HiSCR75) at week 16, was 39%, not much higher than the placebo score of 29% (p=0.3278). HiSCR75 is defined as a ≥75% reduction in total abscess and inflammatory nodule count with no increase in the number of abscesses or tunnels under the skin – hallmark features of HS – relative to baseline. 

Humacyte’s phase II/III tissue graft data prompt talk of a BLA

Humacyte Inc. has posted positive top-line phase II/III results for its Human Acellular Vessel (HAV), a tissue-engineered graft consisting entirely of decellularized extracellular matrix, for vascular trauma repair. The data showed higher rates of patency, a measure of the lack of vascular obstruction, when compared to synthetic graft benchmarks. Results also revealed there were fewer amputations and infections in participants when compared to synthetic graft benchmarks. The data prompted Humacyte to say it plans to file a BLA for HAV for treating urgent arterial repair after extremity vascular trauma when a synthetic graft is not indicated and a saphenous vein is not feasible with the U.S. FDA before 2023 ends.

Precision med startup Actio draws $55M series A investment

“From one to many” is how Actio Biosciences Inc. describes its approach to drug development. The firm emerged with a $55 million series A financing and an eye for biological targets found in both rare and common diseases, starting with TRPV4, a target associated with Charcot-Marie-Tooth disease type 2C (CMT2C) and other bone diseases. The series A was led by Canaan and Droia Ventures, with participation from existing investors Deerfield Management and Ecor1, with new investor Euclidean Capital coming on board. Funds are expected to “get us well into the clinic with our lead program,” said CEO David Goldstein.

Following Hong Kong IPO, Immuneonco looks to be rising star

After raising HKD$320 million (US$40.8 million) in its IPO on the Hong Kong Stock Exchange (HKEX) last week, Immuneonco Biopharmaceuticals Co. Ltd.’s stock has continued to rise, and analysts are expecting the Hong Kong market to pick up after a slack start to the year. The IPO offering for Shanghai-based Immuneonco consisted of 17.2 million shares priced at HKD$18.60 per share. Immuneonco’s stock on the HKEX (HK:1541) was trading at HKD$25.90 per share, up 7.47%, closing at HKD$1.80 on Sept. 12. Although global capital markets took a nosedive in the first half of 2023, Deloitte China predicts that the Hong Kong IPO market will rebound in the second half of 2023 as more Chinese stimulus measures are introduced.

ACIP next step in rolling out updated US COVID-19 vaccines

The U.S. CDC’s Advisory Committee on Immunization Practices (ACIP) will make its recommendations today on the use of monovalent mRNA COVID-19 vaccines the FDA approved and authorized yesterday to target the omicron variant XBB.1.5. A big change in the new approvals of Pfizer Inc./Biontech SE’s Comirnaty and Moderna Inc.’s Spikevax is the number of doses. While both had been approved as two-dose regimens, they are now approved as single-dose vaccines. The Spikevax approval also expanded the age group for people 12 and older, aligning it with the Comirnaty patient population. In addition to the approvals, the FDA authorized the 2023-2024 formula for both vaccines for emergency use in children, ages 6 months through 11 years.

KIW 2023: ‘HLB ready to take 50% of liver cancer market with rivoceranib’

At the second day of the Korea Investment Week (KIW) 2023 conference on Sept. 12 at the Conrad Seoul, HLB Co. Ltd. CEO Jin Yang-gon highlighted how the firm, for over the past 16 years, rose past the public “mockery” from analysts and industry experts when it started anew as a novel drug developer and parted ways from its beginnings as a spinoff in 1975 from the South Korean conglomerate, Hyundai Motor Group. Despite the ridicule, Jin said, the company now stands on the brink of gaining U.S. FDA approval for rivoceranib, and the firm is ready to enter and capture one of the biggest cancer treatment markets worldwide.

BioWorld Insider Podcast – AI: driving drug development from effective to remarkable

Google’s Scott Penberthy joins the podcast for a visionary discussion that scans the horizon for startling changes artificial intelligence will bring to drug development in the relatively near future. Among the gems and eyebrow raisers is talk of dramatic reductions in the time it takes to identify the right molecule for development and how digital clinical trials in the not-too-distant future will substantially shrink study times. This episode also provides a preview of the annual Biofuture conference. Each year, a group of trailblazers, disruptors and forward-thinking executives converge to evaluate and forecast the future of health care. This year, BioWorld is a gold sponsor of the Oct. 4-6 event in New York. If you attend, you'll have the chance to hear panels and join workshops and fireside chats with key opinion leaders like Penberthy.

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