FDA+ roundup: ALS bill would pro­vide $100M an­nu­al­ly to ac­cess po­ten­tial drugs; FDA needs to bet­ter en­force Clin­i­cal­Tri­als.gov re­port­ing, re­searchers say

The House En­er­gy & Com­merce Com­mit­tee be­gan mark­ing up a dozen bills on Wednes­day morn­ing in­clud­ing one that would re­quire the FDA to craft a five-year ac­tion plan for fos­ter­ing the de­vel­op­ment of drugs that im­prove or ex­tend the lives of peo­ple liv­ing with rare neu­rode­gen­er­a­tive dis­eases.

Rare neu­rode­gen­er­a­tive dis­eases, like amy­otroph­ic lat­er­al scle­ro­sis or ALS, have been his­tor­i­cal­ly very dif­fi­cult to treat. But this bi­par­ti­san bill, in­tro­duced by Rep. Mike Quigley (D-IL), will pro­vide $100 mil­lion for each of fis­cal years 2022 through 2026 to help HHS award grants to fa­cil­i­tate ac­cess to in­ves­ti­ga­tion­al drugs that di­ag­nose or treat ALS.

The FDA will award grants to pub­lic and pri­vate en­ti­ties to cov­er the costs of R&D for drugs that di­ag­nose or treat ALS and oth­er se­vere­ly de­bil­i­tat­ing neu­rode­gen­er­a­tive dis­eases, the bill text says.

In ad­di­tion, HHS would have to es­tab­lish the Pub­lic-Pri­vate Part­ner­ship for Neu­rode­gen­er­a­tive Dis­eases be­tween the NIH, FDA, and at least one el­i­gi­ble en­ti­ty (gen­er­al­ly, an in­sti­tu­tion of high­er ed­u­ca­tion or a non­prof­it or­ga­ni­za­tion). The bill, first in­tro­duced last sum­mer, al­so has a com­pan­ion bill in the Sen­ate, in­tro­duced by Chris Coons (D-DE).

FDA needs to bet­ter en­force Clin­i­cal­Tri­als.gov re­port­ing, Yale re­searchers write in JA­MA

De­spite clear in­struc­tions on how and when to re­port clin­i­cal tri­al re­sults, about 60% of tri­als fail to re­port re­sults on time and more than 30% (among al­most 3000 clin­i­cal tri­als with pri­ma­ry com­ple­tion dates be­tween Jan. 2017, and Jan. 2021) have not yet re­port­ed re­sults. That lack of re­port­ing means the FDA needs to beef up its en­force­ment ef­forts, re­searchers from Yale wrote re­cent­ly in a JA­MA view­point.

For its part, the FDA iden­ti­fies po­ten­tial vi­o­la­tions of the law gov­ern­ing the re­port­ing of the re­sults through in­ves­ti­ga­tions and third-par­ty com­plaints, and then at its dis­cre­tion, may is­sue a Pre­lim­i­nary No­tice of Non­com­pli­ance in­form­ing re­spon­si­ble par­ties of po­ten­tial vi­o­la­tions.

Yale re­searchers used FOIA to find that the FDA has on­ly is­sued 58 of its pre­lim­i­nary no­tices of non­com­pli­ance from 2013 through April 29, which they said is “a tiny frac­tion of the thou­sands of FDAAA-ap­plic­a­ble clin­i­cal tri­als iden­ti­fied as non­com­pli­ant with re­sults in­for­ma­tion re­port­ing re­quire­ments as of Jan­u­ary 2021.”

So how can the FDA help? The re­searchers say that in­stead of re­ly­ing on in­con­sis­tent BI­MO in­ves­ti­ga­tions and third-par­ty com­plaints to iden­ti­fy non­com­pli­ant tri­als, the FDA could in­stead use a con­tin­u­al­ly up­dat­ed list of FDAAA 801 prob­lems main­tained by the NIH. In ad­di­tion, FDA should pub­li­cize the no­tices more fre­quent­ly and en­sure that they present clear time­lines for fur­ther en­force­ment ac­tions if re­sults re­main un­re­port­ed.

“The FDA can and should har­ness its en­force­ment tools to en­sure time­ly sub­mis­sion of tri­al re­sults in­for­ma­tion to Clin­i­cal­Tri­als.gov. By do­ing so, the FDA could bring im­por­tant miss­ing re­sults to light and demon­strate its com­mit­ment to pro­tect­ing pa­tients through clin­i­cal tri­al trans­paren­cy,” they wrote.

Re­gen­eron and No­var­tis seek tweaks to ICH’s S12 guid­ance for gene ther­a­pies

No­var­tis, Re­gen­eron and oth­ers are sug­gest­ing tweaks in re­cent­ly re­leased com­ments on a re­cent guid­ance from ICH, known as S12, which deals with the non­clin­i­cal biodis­tri­b­u­tion con­sid­er­a­tions for gene ther­a­pies.

No­var­tis, for in­stance, took is­sue with the fact that vi­ral shed­ding is list­ed as “out of scope” from the S12 guid­ance.

“We be­lieve that it is a missed op­por­tu­ni­ty in not in­clud­ing this top­ic, par­tic­u­lar­ly as some would con­sid­er shed­ding as part of the ‘dis­tri­b­u­tion’ of a gene ther­a­py vec­tor. In ad­di­tion, there ex­ists sig­nif­i­cant health au­thor­i­ty di­ver­gence in opin­ion with re­spect to whether shed­ding should be as­sessed in non­clin­i­cal stud­ies. Please con­sid­er adding shed­ding with­in this guid­ance,” Tim­o­thy MacLach­lan, ex­ec­u­tive di­rec­tor of pre-clin­i­cal safe­ty, and Jes­si­ca Riz­zo, reg­u­la­to­ry pol­i­cy man­ag­er, wrote.

And while the guid­ance sug­gests that non­clin­i­cal BD should be as­sessed pri­or to the ini­ti­a­tion of a clin­i­cal tri­al, No­var­tis won­ders what to do in a sit­u­a­tion where a spon­sor is at­tempt­ing to open an IND with an in­ter­im look on a much longer-term an­i­mal study, where spon­sors would get ad­di­tion­al in­for­ma­tion for the clin­i­cal tri­al from that an­i­mal study.

Re­gen­eron, mean­while, is seek­ing clar­i­ty on the ICH rec­om­men­da­tion for sam­ple col­lec­tion of a core pan­el of tis­sues/bioflu­ids. The New York biotech added:

The guid­ance could be read as rec­om­mend­ing that all of the list­ed tis­sue sam­ples should be col­lect­ed. In­stead, Re­gen­eron pro­pos­es that the guid­ance should rec­om­mend a risk-based ap­proach to guide which sam­ples should be col­lect­ed along with ap­pro­pri­ate jus­ti­fi­ca­tion pro­vid­ed by the de­vel­op­er, based on knowl­edge of the gene ther­a­py prod­uct and its tar­get(s). The same risk-based ap­proach could be used to de­ter­mine which of the col­lect­ed tis­sues would be an­a­lyzed. Do­ing so would al­low de­vel­op­ers to iden­ti­fy those sam­ple tis­sues that can pro­vide ear­ly sci­en­tif­ic ev­i­dence that could in­form fu­ture in-hu­man tri­als.

FDA must prompt­ly pun­ish Min­neso­ta Hos­pi­tal re­searchers, Pub­lic Cit­i­zen says

Pub­lic Cit­i­zen, a non­prof­it con­sumer ad­vo­ca­cy or­ga­ni­za­tion, filed a cit­i­zen pe­ti­tion on Wednes­day with the FDA, call­ing on the agency to take swift en­force­ment ac­tion against sev­er­al Min­neso­ta doc­tors cit­ed in re­cent warn­ing let­ters who con­duct­ed clin­i­cal re­search of dan­ger­ous drugs with­out the prop­er au­tho­riza­tions in place.

Ac­cord­ing to a warn­ing let­ter sent by FDA in Oc­to­ber, doc­tors nev­er filed the ap­pro­pri­ate INDs for the ke­t­a­mine tri­als with the FDA, as re­quired by law, and didn’t write ap­pro­pri­ate pro­to­cols to en­sure that chil­dren and preg­nant women weren’t en­rolled. The tri­als al­so didn’t ex­clude those who were un­der the in­flu­ence of in­tox­i­cants, in whom the use of ke­t­a­mine is cau­tioned.

“A slap-on-the-wrist ap­proach for such non­com­pli­ance that sig­nif­i­cant­ly vi­o­lat­ed the rights of more than 1,700 vul­ner­a­ble hu­man sub­jects and en­dan­gered the health of safe­ty of many of these sub­jects will not suf­fice,” Pub­lic Cit­i­zen wrote.

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.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for 'New Bio­haven' with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the “New Biohaven” post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

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.

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.

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.

Robert Califf, FDA commissioner (via AP Images)

User fees in ac­tion: FDA un­veils new short­ened sup­ple­ment re­view, rare dis­ease pi­lots

Thanks to PDUFA VII, signed into law last Friday by President Joe Biden, the FDA this week unveiled two new industry-friendly pilot programs to advance new rare disease endpoints via additional meetings, and to shorten FDA review times for supplemental apps aimed at unmet medical needs.

The agency this week released eagerly-awaited details behind the shortened pilot, known as the Split Real Time Application Review or STAR pilot program, which will speed up certain FDA reviews of efficacy supplements across all therapeutic areas (thanks to earlier submissions of data), but only for those that propose addressing an unmet medical need.

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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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