Albert Bourla, AP Images

Pfiz­er re­veals de­lays across the board for its clin­i­cal gene ther­a­py pro­grams

At JP Mor­gan 2020, a year af­ter tak­ing the reins at Pfiz­er, Al­bert Bourla claimed the com­pa­ny was poised to be­come a leader in gene ther­a­py. He’s since thrown hun­dreds of mil­lions of dol­lars at those plans, in­clud­ing a $630 mil­lion deal just last month for two of Voy­ager’s AAVs.

Now, it ap­pears Phase III read­outs are fur­ther away than Bourla had hoped.

Set­backs have re­sult­ed in de­lays across all three of Pfiz­er’s clin­i­cal gene ther­a­py pro­grams, the com­pa­ny re­vealed in its Q3 re­sults on Tues­day — in­clud­ing one tri­al which was paused over safe­ty con­cerns.

Pfiz­er’s slam­ming the brakes on its Sang­amo-part­nered Phase III he­mo­phil­ia A study af­ter some pa­tients re­port­ed high lev­els of Fac­tor VI­II, an es­sen­tial blood-clot­ting pro­tein. Nor­mal Fac­tor VI­II lev­els typ­i­cal­ly range from 50% to 100%, and any­thing low­er than 50% could in­di­cate he­mo­phil­ia A. How­ev­er, lev­els above 150% are as­so­ci­at­ed with an in­creased risk of throm­bo­sis, or the for­ma­tion of clots in the blood ves­sels.

Mul­ti­ple pa­tients in Pfiz­er’s he­mo­phil­ia A tri­al had Fac­tor VI­II lev­els over 150%, though the com­pa­ny has not said how many. To date, no pa­tients have ex­pe­ri­enced throm­bot­ic events, and some are be­ing treat­ed with oral an­ti­co­ag­u­lants to re­duce the risk, Pfiz­er said.

The com­pa­ny says it has vol­un­tar­i­ly halt­ed screen­ing and dos­ing in the study — which is more than half-full — un­til it can amend the pro­to­col to pro­vide guide­lines for the man­age­ment of el­e­vat­ed Fac­tor VI­II lev­els.

This isn’t Pfiz­er’s on­ly he­mo­phil­ia set­back. Sep­a­rate­ly, the com­pa­ny said, “re­cent in­ter­ac­tions with the FDA” have led it to de­cide against con­duct­ing in­ter­im analy­ses for both its he­mo­phil­ia A and B stud­ies. In­stead, it will wait for full analy­ses based on at least 50 par­tic­i­pants in the he­mo­phil­ia A study and 40 par­tic­i­pants in the he­mo­phil­ia B study, push­ing out the time­lines for both of the pro­grams.

While Pfiz­er had been an­tic­i­pat­ing read­outs in 2022 for its he­mo­phil­ia B and A pro­grams, it is now look­ing at a Q1 2023 read­out for he­mo­phil­ia B. The com­pa­ny hasn’t pro­vid­ed a new time­line for the he­mo­phil­ia A pro­gram.

That could put Pfiz­er sig­nif­i­cant­ly be­hind its ri­val uniQure, which read out some pos­i­tive Phase III re­sults from a 54-pa­tient tri­al back in June. Over the course of a 52-week pe­ri­od, uniQure’s drug re­duced pa­tients’ an­nu­al­ized rate of bleed­ing re­quir­ing treat­ment by 80% from base­line. That tri­al was briefly put on hold in De­cem­ber fol­low­ing the di­ag­no­sis of HCC in one pa­tient, but reg­u­la­tors gave uniQure the OK to re­sume in April.

uniQure says it plans to sub­mit a BLA in the first quar­ter of next year.

Pfiz­er’s al­so ex­pect­ing a de­lay in its piv­otal DMD study, which is now ex­pect­ed to read out in 2023. The news comes af­ter safe­ty con­cerns led Pfiz­er to scale back that tri­al, cut­ting out pa­tients with cer­tain ge­net­ic mu­ta­tions.

Three cas­es of mus­cle weak­ness, two of which in­clud­ed heart in­flam­ma­tion, forced the tri­al amend­ment. In its Q3 re­sults, Pfiz­er said it’s be­lieved that an im­mune re­sponse against the mi­ni-dy­s­trophin pro­tein caused these cas­es. All pa­tients were treat­ed with high­er dos­es of steroids, and all im­proved with­in a few weeks, though some are still re­cov­er­ing.

“This type of re­ac­tion is a risk po­ten­tial­ly in­her­ent to any gene re­place­ment ther­a­py, and sim­i­lar se­vere ad­verse events re­port­ed in oth­er pro­grams sup­port the no­tion that this is a class ef­fect,” Pfiz­er said.

This isn’t the first time the DMD pro­gram has hit a road­block on its late-stage path, with the com­pa­ny re­port­ing in May that it was meet­ing head­winds from the FDA on the qual­i­ty of its po­ten­cy as­says.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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