Takeda, Sarepta double down on a biotech partner’s platform to engineer better AAV capsids by mapping out their structure

Aravind Asokan and Sapan Shah (StrideBio)

March 16, 2021 08:49 AM EDTUpdated March 19, 02:02 PM

Takeda, Sarepta double down on a biotech partner's platform to engineer better AAV capsids by mapping out their structure

Amber Tong

Senior Editor

When Aravind Asokan and Mavis Agbandje-McKenna first pulled together their respective research at the University of North Carolina-Chapel Hill and the University of Florida on engineering adeno-associated virus vectors and started StrideBio in 2015, the FDA was still two years away from an eventual groundbreaking first approval for an AAV gene therapy.

But even then — and moreso since — preclinical and clinical studies had exposed the glaring room for improvement in the way genes are delivered.

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July 24, 2021 06:00 AM EDT

Weekly

Novartis reshuffles its wild cards; Tough sell for Biogen? Googling proteins; Ken Frazier's new gig; 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.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

July 23, 2021 11:15 AM EDT

Discovery

Google's DeepMind opens its protein database to science — potentially cracking drug R&D wide open

Jason Mast

Editor

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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July 21, 2021 07:17 PM EDTUpdated July 23, 03:30 PM

Pharma

Inside Biogen's scramble to sell Aduhelm: Project 'Javelin' and pressure to ID as many patients as possible

Zachary Brennan

Senior Editor

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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ENDPOINTS CAREERS

Director-Regulatory

Icosavax, Inc.

Seattle, WA, USA

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

July 21, 2021 09:51 AM EDTUpdated July 23, 01:52 PM

R&D

Novartis discards one of its ‘wild card’ drugs after it flops in key study. But it takes one more for the hand

John Carroll

Editor & Founder

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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July 23, 2021 10:31 AM EDTUpdated 03:58 PM

Financing

UPDATED: Three biotechs price hefty IPOs just before the weekend, while a fourth and a SPAC seek spots on Wall Street

Nicole DeFeudis

Editor

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

A handful of biotechs are hitting Wall Street just before the start of the weekend, with three companies — Caribou Biosciences, Sophia Genetics and Absci — all pricing big raises Wednesday and Thursday. Gamma delta T cell-focused IN8bio relaunched its IPO campaign months after postponing it last November, seeking a slightly lower raise. And another SPAC has filed for a public debut.

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Victor Perlroth, Kodiak Sciences CEO

July 23, 2021 10:24 AM EDT

Financing

Kodiak turns down $125M payment from Baker Bros. deal, slashes royalty cap by 55%

Max Gelman

Editor

Following a massive public raise last November, Kodiak Sciences has re-worked a royalty sale agreement with an old partner — and declined new funds in the process.

Kodiak is turning down a planned $125 million payment from Baker Bros. Advisors, according to an SEC filing, cutting short an agreement that saw the biotech hand over a 4.5% stream of royalty sales on its experimental anti-VEGF therapy KSI-301 for retinal vascular diseases. In conjunction with the move, Kodiak is shrinking the royalty cap from just over $1 billion to $450 million.

July 23, 2021 09:49 AM EDT

Pharma

FDA+

EMA rejects FDA-approved Parkinson's drug, signs off on Moderna vaccine use in adolescents ahead of FDA

Zachary Brennan

Senior Editor

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

ENDPOINTS CAREERS

Director, High-Throughput Screening (HTS) Drug Discovery Group

Pliant Therapeutics

South San Francisco, CA, USA

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July 23, 2021 06:58 AM EDT

R&D

Pharma

6 top drugmakers offer perspectives on FDA's new covariates in RCTs guidance

Zachary Brennan

Senior Editor

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Laurent Fischer, Adverum CEO

July 23, 2021 06:25 AM EDT

R&D

Cell/Gene Tx

Adverum faces murky future after review turns up deeper safety issues for gene therapy

Jason Mast

Editor

Three months after revealing that a patient lost significant vision in one eye after receiving its experimental gene therapy, Adverum announced it found the safety issues were more widespread: Five of 12 patients who received a high dose of the therapy saw “similar clinically-relevant events.”

Three required surgery on their treated eye. And all 12 are being recommended “aggressive immunomodulatory treatments” to prevent further injury.

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