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April 14, 2022 09:36 AM EDTUpdated 05:05 PM
Cell/Gene Tx
In Focus

'Em­pa­thy and love were not shown’: The sto­ry be­hind Taysha’s lay­offs, one of dozens of biotechs suf­fer­ing this year’s bear mar­ket

Kyle LaHucik

Associate Editor

A biotech launched by former AveXis leaders was on a mission to treat multiple rare diseases with new gene therapies. They speedily assembled a team and touted their fast ascent from seed to IPO, in a mere six-month window in 2020, to repeat the playbook they created with the FDA-approved SMA drug Zolgensma.

Flush with a pipeline of more than 25 programs, Taysha Gene Therapies worked in concert with UT Southwestern to take the university’s gene therapy research into the clinic with the ambition of treating monogenic diseases of the central nervous system and pediatric indications. About half a dozen have made it into human trials, with the lead program on track for registration pathway feedback mid-year.

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May 24, 2022 08:49 AM EDTUpdated 03:54 PM
R&D
In Focus

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

Nicole DeFeudis

Editor

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Ted White, Verrica Pharmaceuticals CEO
May 25, 2022 06:33 AM EDT
FDA+

'Hands may be tied': FDA slaps Ver­ri­ca with 3rd CRL due to prob­lems with con­tract man­u­fac­tur­er

Amber Tong

Senior Editor

The FDA has rejected Verrica Pharmaceuticals’ skin disease treatment for a third time — and once again the contract manufacturer is to blame.

The biotech emphasized that the only deficiency in the complete response letter is related to a general reinspection of the CMO, Sterling Pharmaceuticals, and has nothing to do specifically with its drug-device; the rest of the NDA is good to go.

CEO Ted White said the company is “extremely disappointed,” but will keep working toward approval.

Mihael Polymeropoulos, Vanda Pharmaceuticals CEO
May 24, 2022 02:29 PM EDT
FDA+

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Zachary Brennan

Senior Editor

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

May 24, 2022 01:29 PM EDT
Coronavirus
Law

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

Zachary Brennan

Senior Editor

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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May 24, 2022 12:48 PM EDT
Pharma
Marketing

Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

Beth Snyder Bulik

Senior Editor

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Michael Corbo, Pfizer CDO of inflammation & immunology
May 24, 2022 10:37 AM EDTUpdated 01:33 PM
R&D

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Amber Tong

Senior Editor

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Robert Califf (Michael Brochstein/Sipa USA via AP Images)
May 24, 2022 06:56 AM EDT
China
FDA+

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Zachary Brennan

Senior Editor

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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David Mazzo, Caladrius Biosciences CEO
May 24, 2022 06:42 AM EDTUpdated 01:28 PM
R&D
Cell/Gene Tx

Blam­ing Covid dis­rup­tion, sup­ply short­ages, mi­cro­cap biotech hits the brakes on cell ther­a­py tri­al

Amber Tong

Senior Editor

More than two years into the Covid-19 pandemic, we’re not done seeing its disruption of biotech.

That’s according to Caladrius execs, who cited the pandemic as one reason for suspending its Phase IIb trial of a cell therapy as a treatment for coronary microvascular dysfunction (CMD). The company plans to go ahead on an interim analysis before deciding what to do with the program, which was built on a licensing deal with Shire in 2018.

May 23, 2022 02:23 PM EDT
Pharma

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Zachary Brennan

Senior Editor

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

'Em­pa­thy and love were not shown’: The sto­ry be­hind Taysha’s lay­offs, one of dozens of biotechs suf­fer­ing this year’s bear mar­ket

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

'Hands may be tied': FDA slaps Ver­ri­ca with 3rd CRL due to prob­lems with con­tract man­u­fac­tur­er

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Blam­ing Covid dis­rup­tion, sup­ply short­ages, mi­cro­cap biotech hits the brakes on cell ther­a­py tri­al

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states