Astellas snags a gene therapy upstart with $3B buyout bid — inspiring forecasts of more M&A ahead

Image: Associated Press

December 2, 2019 08:15 PM ESTUpdated 09:57 AM

Astellas snags a gene therapy upstart with $3B buyout bid — inspiring forecasts of more M&A ahead

John Carroll

Editor & Founder

Astellas has moved in to buy a biotech upstart with a new approach to developing a gene therapy for Duchenne muscular dystrophy.

The Japanese pharma player has struck a deal to acquire Audentes $BOLD for about $3 billion, or $60 a share — a 110% premium over the $28.61 close on Monday. And Astellas is signaling its plans to add more gene therapy deals around Audentes, which it is plugging into its global research organization.

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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.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

October 22, 2021 08:46 AM EDTUpdated 09:47 AM

R&D

No, scientists are not any closer to pig-to-human transplants than they were last week

Jason Mast

Editor

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

October 22, 2021 08:00 AM EDTUpdated 03:32 PM

FDA+

UPDATED: Agenus calls out FDA for playing favorites with Merck, pulls cervical cancer BLA at agency's request

Zachary Brennan

Senior Editor

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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Account Supervisor, Healthcare

New York, NY, USA

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October 21, 2021 11:09 AM EDTUpdated 3 hours ago

FDA+

How to collect and submit RWD to win approval for a new drug indication: FDA spells it out in a long-awaited guidance

Zachary Brennan

Senior Editor

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Marty Duvall, Oncopeptides CEO

October 22, 2021 10:44 AM EDT

Pharma

FDA+

Oncopeptides stock craters as it pulls cancer drug Pepaxto from the market

Zachary Brennan

Senior Editor

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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October 22, 2021 10:39 AM EDT

FDA+

Coronavirus

Pfizer pitches its Covid-19 vaccine for younger children ahead of adcomm next week

Zachary Brennan

Senior Editor

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

David Lockhart, ReCode Therapeutics CEO

October 21, 2021 08:13 AM EDTUpdated 14 hours ago

Financing

Cell/Gene Tx

Pfizer throws its weight behind LNP player eyeing mRNA treatments for CF, PCD

Amber Tong

Senior Editor

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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October 21, 2021 07:07 AM EDT

Deals

Pharma

Bristol Myers pledges to sell its Acceleron shares as activist investors circle Merck's $11.5B buyout — report

Amber Tong

Senior Editor

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

David Livingston (Credit: Michael Sazel for CeMM)

October 20, 2021 03:56 AM EDT

People

Renowned Dana-Farber scientist, mentor and biopharma advisor David Livingston has died

John Carroll

Editor & Founder

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.