The first pa­tient has been treat­ed un­der the con­tro­ver­sial 'right to try' law

A pa­tient di­ag­nosed with an ag­gres­sive form of brain can­cer be­came the first per­son in the US to ac­cess an ex­per­i­men­tal treat­ment un­der Right to Try. Signed in­to law by Pres­i­dent Don­ald Trump in May 2018, it has been tout­ed as a new way to help peo­ple with ter­mi­nal ill­ness­es and few op­tions, al­though un­til now, no pa­tients had ever used it.

ERC-USA and the Uni­ver­si­ty of Cal­i­for­nia, Irvine ini­ti­at­ed treat­ment with the com­pa­ny’s in­ves­ti­ga­tion­al com­pound ERC-1671 — known as Glio­vac in Eu­rope — and which is in Phase 2 clin­i­cal tri­als in the US. The pa­tient’s treat­ment with ERC-1671 be­gan at the uni­ver­si­ty in late No­vem­ber 2018.

The pa­tient re­sort­ed to Right to Try af­ter fail­ing to qual­i­fy for en­roll­ment in the on­go­ing tri­al. But rather than work around the agency that may even­tu­al­ly ap­prove the ex­per­i­men­tal treat­ment, the com­pa­ny said in Au­gust that it in­formed the FDA that it in­tend­ed to make the ex­per­i­men­tal treat­ment avail­able to this one pa­tient. FDA ac­knowl­edged ac­cep­tance of the com­pa­ny’s no­ti­fi­ca­tion on 13 Ju­ly, ERC said.

Oth­er com­pa­nies, in­clud­ing Ther­a­peu­tics So­lu­tions In­ter­na­tion­al, have an­nounced their in­ten­tions to use Right to Try, though ac­cord­ing to Gold­wa­ter In­sti­tute, no oth­er pa­tients have yet to use the new law.

The law has proven to be a thorn in the side of FDA, which has said it will work to im­ple­ment it in a man­ner con­sis­tent with con­gres­sion­al in­tent and with FDA’s pub­lic health mis­sion, but the agency still has its own process for help­ing ter­mi­nal­ly ill pa­tients re­ceive ex­per­i­men­tal treat­ments, known as ex­pand­ed ac­cess. FDA grants about 99% of the ex­pand­ed ac­cess re­quests it re­ceives.

Ac­cord­ing to Bio­Cen­tu­ry, FDA’s On­col­o­gy Cen­ter of Ex­cel­lence is al­so work­ing on a new ini­tia­tive, known as Pro­ject Fa­cil­i­tate, un­der which the agency will pro­vide a tele­phone num­ber that pa­tients and physi­cians seek­ing ex­pand­ed ac­cess to an ex­per­i­men­tal treat­ment can call. FDA staff will an­swer calls and fill out the form re­quired to ap­ply for a sin­gle-pa­tient IND re­quest. The pa­per­work will be for­ward­ed to the man­u­fac­tur­er. A pi­lot ver­sion of this ini­tia­tive is ex­pect­ed to launch in the first half of 2019.

First pub­lished here and syn­di­cat­ed in part­ner­ship with RAPS.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

What's fair? New ICER re­port shows pay­ers gen­er­al­ly en­sur­ing fair ac­cess to drugs

The nonprofit Institute for Clinical and Economic Review on Wednesday released a new report highlighting the ways in which payers are generally ensuring fair access to prescription drugs, even when based on a set of criteria set by the nonprofit.

While noting the lack of transparency hindered the report’s results, ICER said that the “great majority” of payer policies in the formularies evaluated are structured in a way to support many key elements of how ICER defines “fair access.”

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

GSK 'Target the Future' Ad Campaign

Glax­o­SmithK­line's friend­ly ‘shark tank’ crowd­sources ideas for mul­ti­ple myelo­ma, kicks off big­ger push

GlaxoSmithKline is inviting everyone to its friendly shark tank. Its “Think Tank” challenge launching today aims to gather the best pitches for ideas in multiple myeloma with a final pitch-off “Shark Tank” TV show-style finish next year.

The innovation contest kicks off GSK’s bigger “Target the Future” unbranded campaign to advance innovation and awareness in multiple myeloma.

“We have a good sense of where the unmet need lies and what tools may be welcomed by this community, and we’ll continue to do that as part of this program, but the ‘Think Tank’ kickoff is to bring new ideas to the table — things that come from a more grassroots perspective than a large pharma perspective,” Christine Roth, senior VP and global head of oncology at GSK, said.

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Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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