The long-anticipated top-line phase IIa study results for Morphic Therapeutic Inc.’s oral alpha 4 beta 7 integrin inhibitor, MORF-057, have surpassed even the company’s own expectations, with a significant decline in disease activity seen in moderate to severe ulcerative colitis (UC) patients combined with a safety profile consistent with phase I findings. The 35 patients in the main cohort of the open-label multicenter phase IIa study – called Emerald 1 – experienced a statistically significant 6.4-point drop (p=0.002) from baseline in the Robarts Histopathology Index (RHI) Score, a measure of histological disease activity, at week 12. At midday on April 25, Morphic’s stock (NASDAQ:MORF) was trading down by 7%.

US FDA turns to patients to deepen understanding of long COVID

As it requested, the U.S. FDA is getting an earful today as people with long COVID and their caregivers share their experiences with the condition that has no approved treatment and is not that well understood, given its range of symptoms that vary from person to person. Among those symptoms, ongoing brain fog and fatigue ranked as the most common in an audience poll the agency took as it opened a public meeting on patient-focused drug development for long COVID. One panelist, 28-year-old Katie Drackert who has had her dancing career sidelined by long COVID, compared the fatigue with a phone that immediately drops to 10% of battery power after being charged all night.

Newco News: Ventoux Biosciences takes aim at Dupuytren’s disease

Dupuytren’s disease is often referred to as “the most common crippling hand condition that people have never heard of,” but Kurt Harrington is on a mission to change that. A seasoned biotech and pharma consultant, Harrington has had the condition himself for over a decade and, acutely aware of the lack of available treatment options, has founded Ventoux Biosciences Inc. in order to bring additional therapies forward.

Swiss biotech navigates the downturn as funding drops 60%

Swiss biotech firms raised CHF1.3 billion (US$1.5 billion) in equity and debt financing in 2022, a fall of 60% from the previous year’s total of over CHF3.3 billion. But it’s still ahead of historic pre-pandemic levels of funding. In Switzerland, as elsewhere, the pandemic-fueled funding bonanza is well and truly over, but nonetheless the sector remains resilient, according to the Swiss Biotech Report 2022, which the Swiss Biotech Association and EY produced. It was published on the annual Swiss Biotech Day event.

China greenlights Janssen’s nasal spray antidepressant for MDD

Xian Janssen Pharmaceutical Ltd. obtained marketing approval in China for Spravato (esketamine hydrochloride nasal spray) in combination with an oral antidepressant to treat depressive symptoms in adults with major depressive disorder (MDD) with acute suicidal ideation or behavior. Unlike existing antidepressants on the market, which can take weeks to take effect, the newly approved nasal spray shows effects right away. “Patients with depression need more treatment options to help them effectively alleviate their symptoms, and it is especially important for depressed patients with acute suicidal ideation or behavior,” said Li Zili, vice president and head of Asia Pacific R&D at Janssen.

Questions arise about nonsilent effect of synonymous mutations

Synonymous or silent mutations do not change the sequence of the protein that they encode. With some exceptions, they do not trigger any effect. Last year, however, a study by researchers from the University of Michigan tried to refute this concept after finding that they altered the protein function. But breaking dogmas can have answers. A group of scientists from various institutions has found that this work could have a method error. “I was aware of the paper from the day it was published because it made a big splash on social media,” Craig Kaplan, an associate professor of the Department of Biological Sciences at the University of Pittsburgh and the lead author of this group of scientists, told BioWorld. “I was really surprised by the results. And upon looking at the methods it was immediately clear to me that there was an experimental design flaw.”

The BioWorld Insider Podcast: Better times ahead for the biopharma sector? Could be, the new numbers say

It used to be a lot easier to find partners. After two years of strong dealmaking, the volume and value of partnerships slid in the first quarter of 2023. M&As also are down compared to prior years. But there are some reasons to be cheerful. Looking at first quarter numbers and surveying the big picture on this BioWorld Insider podcast are Karen Carey, BioWorld’s managing editor and senior data analyst, along with Tim Shannon, a general partner at Canaan. Carey just wrote about biopharma deals and completed M&As in the first quarter. Shannon, who’s been with Canaan since 2009, has a new fund sporting $850 million in new capital, including an oversubscribed $650 million fund for seed and series A financings. They both offer their insights into the first quarter’s numbers and what may lie ahead.

Also in the news

Adcentrx, Aditxt, Albatroz, Alkermes, Alx, Ani, Apellis, Applied, Arca, Assertio, Astrazeneca, Avidity, Belief, Biomarin, Bluebird, Bridge, Citius, Cue, Daewoong, Enanta, Faron, Foghorn, Forge, GRI, Guardant, HDL, Ideaya, I-Mab, Immunos, In8bio, Ionis, Iveric, Kancera, Kineta, Kronos, Krystal, Lantheus, Lixte, Maat, Maia, Maxwell, Medigene, Mina, Moderna, Neurona, Newamsterdam, Novo Nordisk, Nuvox, Omers, Oncoinvent, Pharma Two B, Point, Sanofi, Spectrum, Stada, Tracon, Unity, VBI, Vedanta, Vyant, Windtree, Wren, Xbrane