The hospital’s gone now but across the street from it, there used to be a clinic, a beat-up thing with peeling paint that, in a past life, a past city, had been a bank. John Sundy was a medical student training at the hospital, in downtown Philadelphia. He walked in and saw a crowd of patients in all manner of autoimmune conditions waiting to see a tan, distinguished-looking man in a white lab coat: well-known rheumatologist Ralph DeHoratius.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,000+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN