Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial — but another safety setback mars results

May 15, 2020 08:02 AM EDTUpdated 09:47 AM

Pfizer gene therapy ups levels of dystrophin expression in Duchenne trial — but another safety setback mars results

John Carroll

Editor & Founder

Pfizer’s gene therapy group is back up at bat with a fresh update of data from its Phase Ib study for a gene therapy intended as a once-and-done treatment for Duchenne muscular dystrophy. And they had some good news, and some bad news, to report.

The good news was primarily in the dystrophin expression they tracked among the now 9 evaluable patients in their first clinical foray for PF-06939926.

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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 16, 2021 06:00 AM EDT

Weekly

Covid-19 vaccine boosters earn big thumbs up, but Moderna draws ire over world supply; What's next for Merck’s Covid pill?; The C-suite view on biotech; and more

Amber Tong

Senior Editor

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.

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October 15, 2021 10:21 AM EDTUpdated 11:05 AM

R&D

Novartis development chief John Tsai: 'We go deep in the new platforms'

John Carroll

Editor & Founder

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

October 14, 2021 10:55 AM EDTUpdated October 15, 03:10 PM

R&D

AstraZeneca oncology R&D chief Susan Galbraith: 'You're going to need orthogonal combinations'

John Carroll

Editor & Founder

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

October 14, 2021 11:07 AM EDTUpdated 11:32 AM

Startups

A star Stanford professor leaves his lab for a startup out to remake psychiatry

Jason Mast

Editor

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

October 15, 2021 04:03 PM EDT

Pharma

FDA+

Roche's Tecentriq crosses the finish line first in adjuvant lung cancer, potentially kicking off gold rush

Kyle Blankenship

Managing Editor

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Susan Galbraith speaking at Endpoints News' virtual EUBIO21 summit

October 15, 2021 07:25 AM EDT

R&D

Imfinzi/tremelimumab combo scores AstraZeneca another OS win — this time in liver cancer

Amber Tong

Senior Editor

Is the tide turning on AstraZeneca’s battered PD-L1/CTLA4 combo?

A single priming dose of the experimental tremelimumab, followed by Imfinzi every four weeks, beat Nexavar (sorafenib) in helping a group of liver cancer patients live longer in a Phase III study, the company reported, meeting the primary endpoint.

Specifically, the two drugs extended overall survival for patients with unresectable hepatocellular carcinoma who had not received prior systemic therapy and were not eligible for localized treatment.

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October 13, 2021 12:09 PM EDTUpdated 02:19 PM

R&D

FDA+ roundup: Marks on Woodcock's tenure as acting commissioner; FDA leaders offer perspective on barriers to diversity in research

Zachary Brennan

Senior Editor

CBER director Peter Marks praised Janet Woodcock’s work as acting FDA commissioner, and while noting that Biden needs to nominate someone to fill the role permanently by Nov. 16, he said he has “no idea” when that actually might occur.

“Dr. Woodcock has been at the agency for over three decades and she, during that time, has proven herself to be a remarkably capable manager,” Marks said at the Alliance for Regenerative Medicine’s meeting Tuesday. “And she’s been managing as if she’s commissioner, unlike some previous acting [commissioners] who are afraid to actually do things. She doesn’t appear to be afraid to do things. I have not felt any different now from when we had a commissioner in place,” he added.

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Samarth Kulkarni, CRISPR CEO

October 12, 2021 04:01 PM EDTUpdated October 13, 08:41 AM

R&D

Cell/Gene Tx

CRISPR Therapeutics claims safety advantage in first big look at off-the-shelf CAR-T data, but durability in question

Jason Mast

Editor

CRISPR Therapeutics CEO Samarth Kulkarni thinks his company might have built the safest CAR-T therapy yet.

The gene editing biotech announced the first major batch of data from its off-the-shelf CAR-T program, showing that 58% of the 26 large B-cell lymphoma patients who received the therapy saw their tumors shrink and that 38% had no signs of cancer whatsoever.

Those response rates, outside experts say, are broadly in the range seen in trials for autologous CAR-T therapies such as Novartis’s Kymriah and Gilead’s Yescarta, bolstering the chance that off-the-shelf could eventually make the benefits of such therapies far more widely available. Uptake of the first-generation of CAR-Ts continues to be limited by the cost, time and infrastructure required to handle each person’s cells.

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