A small biotech looking to develop a gene therapy for a rare condition that can cause color or legal blindness ran into some significant safety trouble in a Phase I/II dose-escalation study.

Applied Genetic Technologies Corporation, also known as AGTC, reported Thursday afternoon that three of five children in the study’s highest dose cohort experienced severe inflammation one month after dosing. The events were concerning enough to be categorized as suspected unexpected serious adverse reactions, or SUSARs.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Mirati Therapeutics’ path to a second drug approval will likely have to wait. The San Diego biotech company said Wednesday that its investigational lung cancer drug failed a Phase III trial testing it in combination with Bristol Myers Squibb’s Opdivo.

The drug, sitravatinib, and Opdivo weren’t better than the chemo drug docetaxel at keeping patients alive, Mirati said in a press release. The spectrum-selective kinase inhibitor missed the primary goal of overall survival in patients with second- or third-line advanced non-squamous, non-small cell lung cancer.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

As Novo Nordisk and Pfizer disclose some data on their oral weight loss drugs in Phase III and II, respectively, Eli Lilly is beefing up its stance in the obesity field with three late-stage clinical trials of its next-generation GLP-1 agonist orforglipron.

The moves, disclosed in updates to the federal clinical trials database this week, put the Indianapolis drugmaker ahead of Pfizer, whose science chief has said the company will “cherry-pick” which of its mid-stage candidates to take deeper into the clinic after data late this year or early next.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN