April 12, 2021 09:05 AM EDT
Cell/Gene Tx
FDA+

Gene ther­a­pies for neu­rode­gen­er­a­tive dis­eases: Pfiz­er, No­var­tis, Bio­gen and oth­ers seek more from FDA

Zachary Brennan

Senior Editor

Top biopharma companies are calling on the FDA to provide more context and examples, particularly on early interactions with the agency, as part of the companies’ feedback on draft guidance from January related to developing gene therapies to treat neurodegenerative diseases.

The 15-page draft guidance outlines various considerations on manufacturing, preclinical and clinical trials for gene therapies targeting amyotrophic lateral sclerosis, Alzheimer’s disease and other neurodegenerative disorders that are notoriously difficult to treat.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

SPONSORED
May 10, 2021 06:00 AM EDT

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Parexel

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)
May 11, 2021 07:01 AM EDTUpdated 11:11 AM
Financing
R&D

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

John Carroll

Editor & Founder

Jason Mast

Editor

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,200+ biopharma pros reading Endpoints daily — and it's free.

May 12, 2021 11:55 AM EDT
Pharma
FDA+

FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

Zachary Brennan

Senior Editor

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

May 12, 2021 06:50 AM EDTUpdated 09:42 AM
FDA+
Manufacturing

A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

Zachary Brennan

Senior Editor

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Distribution of Moderna's Covid-19 Vaccine (Photo by Paul Sancya - Pool/Getty Images)
May 11, 2021 03:24 PM EDT
FDA+
Opinion

Opin­ion: Ado­les­cents can wait. The US needs to start do­nat­ing Covid-19 vac­cines to needy coun­tries now.

Zachary Brennan

Senior Editor

Now that the US is swimming in Covid-19 vaccines and the supply has officially eclipsed the demand, it’s time for America to lead the world and start shipping these excess doses to countries that desperately need them.

Unlike the IP waiver at the World Trade Organization, which Biden now supports and will likely take years to translate into actual shots in arms, the US could easily donate just a tiny fraction of the more than 60 million doses of Pfizer, Moderna and J&J vaccines sitting on American shelves right now.

May 12, 2021 11:18 AM EDT
Pharma

Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

Zachary Brennan

Senior Editor

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,200+ biopharma pros reading Endpoints daily — and it's free.

May 12, 2021 10:59 AM EDTUpdated 11:12 AM
FDA+

UP­DAT­ED: Feds charge an­oth­er CRO staffer with fak­ing da­ta in a Glax­o­SmithK­line pe­di­atric asth­ma study

Max Gelman

Associate Editor

A Florida woman has been indicted as part of a clinical trial fraud scheme over a GlaxoSmithKline pediatric asthma study, the Justice Department announced Tuesday, the latest development in a case where three individuals have already pleaded guilty.

Jessica Palacio was charged with participating in a plot to falsify medical records, giving off the appearance that trial participants were making their scheduled visits to a Miami CRO and taking an experimental asthma medication as required. Palacio was also charged with lying to FDA investigators about her conduct.

Chris Garabedian (Xontogeny)
May 12, 2021 07:29 AM EDT
Financing

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

Josh Sullivan

Associate Editor

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,200+ biopharma pros reading Endpoints daily — and it's free.

Stephen Squinto, Gennao Bio CEO (Gennao)
May 12, 2021 06:39 AM EDTUpdated 06:53 AM
People
Financing

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

Josh Sullivan

Associate Editor

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Gene ther­a­pies for neu­rode­gen­er­a­tive dis­eases: Pfiz­er, No­var­tis, Bio­gen and oth­ers seek more from FDA

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

Opin­ion: Ado­les­cents can wait. The US needs to start do­nat­ing Covid-19 vac­cines to needy coun­tries now.

Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

UP­DAT­ED: Feds charge an­oth­er CRO staffer with fak­ing da­ta in a Glax­o­SmithK­line pe­di­atric asth­ma study

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er