UniQure gets another shot with hemophilia gene therapy after FDA lifts clinical hold; Neoleukin’s IL-2 hopeful also cleared

Matt Kapusta, uniQure CEO (via website)

April 26, 2021 08:48 AM EDTUpdated May 3, 01:13 PM

UniQure gets another shot with hemophilia gene therapy after FDA lifts clinical hold; Neoleukin's IL-2 hopeful also cleared

Kyle Blankenship

Managing Editor

Months after a disastrous clinical hold on its hemophilia B gene therapy shut down development, uniQure had reason to believe its drug wasn’t to blame for the single case of liver cancer that halted the study. After presenting what it had to the FDA, the biotech will get another shot — and it’s got topline data teed up and ready to go.

Weeks after the biotech determined its gene therapy was likely not linked to a single case of liver cancer, the FDA has lifted a five-month clinical hold on uniQure’s etranacogene dezaparvovec, the company confirmed Monday.

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Biotech Halftime Report: After a bumpy year, is biotech ready to rebound?

Brian Abrahams

Co-Head of Biotechnology Research

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

October 18, 2021 07:01 AM EDTUpdated 04:19 PM

Pharma

FDA+

UPDATED: Boehringer nabs FDA's first interchangeability designation for its Humira competitor — but will it matter?

Zachary Brennan

Senior Editor

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

October 17, 2021 05:42 PM EDTUpdated 18 hours ago

R&D

Biogen hit by ALS setback with PhIII failure for tofersen — but follows a familiar strategy highlighting the positive

John Carroll

Editor & Founder

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.

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Reshma Kewalramani, Vertex CEO (YouTube)

October 18, 2021 07:30 AM EDTUpdated 01:29 PM

R&D

Cell/Gene Tx

Vertex gets much-needed win with ‘extraordinary’ first patient results on potential diabetes cure

Jason Mast

Editor

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.

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Jeffrey Nau, Oyster Point Pharma CEO

October 18, 2021 10:45 AM EDT

Pharma

FDA+

FDA OKs an inhaled version of smoking cessation drug Chantix — for a common eye disease

Paul Schloesser

Editorial Intern

Oyster Point Pharma now has its first FDA-approved product — Tyrvaya. And the biotech has taken a unique route to get there by using an old drug with a storied past.

The New Jersey biotech announced this morning that the FDA has approved their nasal spray product for dry eye disease on Friday — the first nasal spray to be approved for the disease. The product’s active ingredient is 0.03 mg of varenicline, also known as smoking cessation aid Chantix.

October 18, 2021 10:35 AM EDT

FDA+

Omeros plunges deeper after FDA rejects rare disease drug, asking for more information