PTC offers its best case for a controversial Duchenne MD PhIII. But key failures spark fresh doubts of success at FDA

June 21, 2022 11:22 AM EDTUpdated June 23, 03:36 AM

PTC offers its best case for a controversial Duchenne MD PhIII. But key failures spark fresh doubts of success at FDA

John Carroll

Editor & Founder

PTC Therapeutics’ years-long effort to push its controversial Duchenne MD drug Translarna into the US has just run into its latest setback.

The biotech reported a mixed set of data from its confirmatory study of the drug, with a list of key failures that at least one high profile analyst believes aren’t likely to win over skeptics at the FDA.

The biotech’s execs were careful to spotlight the upbeat aspects of the data, which they say lays a straight path to their next FDA pitch for treating nonsense mutation DMD. And RBC’s Brian Abrahams believes that should keep it on the European market, where it got an early launch on failed trial data. But the analyst raised doubts over the bad data PTC divulged Tuesday morning.

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June 27, 2022 07:00 AM EDT

R&D

Scoop: Boehringer quietly shutters a PhII for one of its top drugs — now under review

Kyle LaHucik

Associate Editor

John Carroll

Editor & Founder

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Lina Gugucheva, NewAmsterdam Pharma CBO

June 28, 2022 08:00 AM EDT

Deals

Pharma group bets up to $1B-plus on the PhIII resurrection of a once dead-and-buried LDL drug

John Carroll

Editor & Founder

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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June 28, 2022 08:37 AM EDTUpdated 08:56 AM

R&D

Pharma

Scoop: Roche scraps one of two schizophrenia PhII trials after failing the primary endpoint

Kyle LaHucik

Associate Editor

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on clinicaltrials.gov. Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

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ENDPOINTS CAREERS

Director of Quality Assurance

ImmunityBio

Dunkirk, NY, USA

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June 28, 2022 07:17 AM EDT

Pharma

In Focus

How prepared is biopharma for the cyber doomsday?

Amber Tong

Senior Editor

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Merdad Parsey, Gilead CMO

June 28, 2022 09:12 AM EDT

R&D

Pharma

Four months after CRL due to contaminant worries, Gilead returns to FDA for next-gen HIV drug

Paul Schloesser

Associate Editor

Just shy of four months ago, Gilead’s next-gen HIV drug candidate lenacapavir got hit with a CRL over CMC issues involving the type of vials planned for use. Now, the pharma is headed back to the FDA for round two.

Gilead announced Monday afternoon that it had refiled its NDA submission filled with new CMC data after the FDA essentially balked at borosilicate glass vials, originally used for the non-oral form of lenacapavir. The drug candidate, which recently won a positive opinion from Europe’s CHMP, is being developed for HIV-1 infection “in heavily treatment-experienced (HTE) people with multi-drug resistant (MDR) HIV-1 infection.”

Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

June 28, 2022 07:47 AM EDT

R&D

Roivant chops sickle cell gene therapy, dermatology drugs to focus on 'higher value projects'

Amber Tong

Senior Editor

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

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June 28, 2022 08:52 AM EDT

Deals

Years after linking arms with Bristol Myers and both Mercks, Sutro finds its latest partner in Tokyo

Kyle LaHucik

Associate Editor

Astellas and Sutro Biopharma are linking arms on a new field of antibody-drug conjugates that they hope will improve upon existing cancer immunotherapies.

The Tokyo pharma will dole out $90 million in cash for the collaboration, the companies said Monday afternoon. That upfront payment will extend the South San Francisco biotech’s runway from late 2023 into the first half of 2024, Cowen analysts noted.

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Laurence Reid, Decibel CEO

June 28, 2022 07:15 AM EDTUpdated 07:26 AM

R&D

Still in preclinical testing for ear gene therapies, Decibel touts small snapshot of chemo-induced hearing loss drug

Max Gelman

Senior Editor

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

June 28, 2022 07:00 AM EDT

People

Startups

Longtime biotech leader Pearl Huang takes the reins as CEO of Novartis-backed upstart

Tyler Patchen

News Reporter

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

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