De­spite a PhI­II fail, Bio­gen push­es ALS drug tofersen for­ward with new analy­sis in move echo­ing Aduhelm

In a move rem­i­nis­cent of its ad­u­canum­ab de­vel­op­ment plans, Bio­gen is tak­ing steps Fri­day to try to paint a failed Phase III in a pos­i­tive light — this time for ALS.

The big bio­phar­ma re­leased a new analy­sis com­bin­ing Phase III da­ta with re­sults from an open-la­bel ex­ten­sion on the Io­n­is-part­nered an­ti­sense drug tofersen. Bio­gen says the drug proved more ef­fec­tive for pa­tients who start­ed tak­ing it ear­li­er in their dis­ease, with the new analy­sis sug­gest­ing a clin­i­cal func­tion ben­e­fit and longer sur­vival times.

But it re­mains un­clear how per­sua­sive these analy­ses will be at the FDA, giv­en tofersen did not achieve its pri­ma­ry Phase III goal.

Back in Oc­to­ber, Bio­gen and Io­n­is re­port­ed tofersen did not sig­nif­i­cant­ly im­prove the func­tion­al and neu­ro­log­ic de­cline of pa­tients af­ter 28 weeks of treat­ment, ob­serv­ing very lit­tle dif­fer­ence in re­sponse be­tween the drug and place­bo groups. The p-val­ue came in at an abysmal p=0.97, equat­ing to a 3% chance that the po­ten­tial ef­fects seen in the tri­al were drug-re­lat­ed and not due to chance.

On Fri­day, how­ev­er, Bio­gen said the pooled Phase III and open-la­bel ex­ten­sion da­ta are proof the drug ac­tu­al­ly works. The com­pa­nies claimed to see ben­e­fits in clin­i­cal func­tion, res­pi­ra­to­ry func­tion, mus­cle strength and qual­i­ty of life.

Tofersen is be­ing eval­u­at­ed in a ge­net­ic sub­set of ALS pa­tients — those with mu­ta­tions in a gene known as SOD1. The ALS As­so­ci­a­tion es­ti­mates that 10% to 20% of pa­tients with fam­i­ly his­to­ries of ALS pos­sess such mu­ta­tions. SOD1-ALS al­so makes up an­oth­er 1% to 2% of pa­tients with­out an ALS fam­i­ly his­to­ry.

The part­ners are al­so ar­gu­ing that the new da­ta shed light on a po­ten­tial ef­fect on ALS bio­mark­ers, of which there are none cur­rent­ly proven to be linked. Re­search to find bio­mark­ers has cen­tered around lev­els of neu­ro­fil­a­ment and oth­er pro­teins, with the the­o­ry be­ing that low­er­ing such el­e­ments is cor­re­lat­ed to a dis­ease-mod­i­fy­ing ef­fect.

Here, the com­pa­nies said the analy­sis showed re­duc­tions in SOD1 and plas­ma neu­ro­fil­a­ment both in pa­tients who start­ed tak­ing tofersen ear­li­er and as pre­vi­ous­ly ad­min­is­tered. It’s part of the “to­tal­i­ty of ev­i­dence” Bio­gen is ar­gu­ing shows tofersen is more ef­fec­tive the ear­li­er pa­tients re­ceive it.

As Bio­gen and Io­n­is ap­proach the FDA with their ap­pli­ca­tion, the sim­i­lar­i­ties to the Alzheimer’s drug Aduhelm — and the fi­as­co that en­sued post-ap­proval — abound. In that in­stance, Bio­gen halt­ed two Phase III stud­ies ear­ly due to fu­til­i­ty, but then re­versed course and said it saw an ef­fect in one of the tri­als af­ter more analy­ses were con­duct­ed.

Clar­i­fi­ca­tion: This ar­ti­cle has been up­dat­ed to clar­i­fy the na­ture of Bio­gen’s pooled da­ta analy­sis. 

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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Ab­bott pumps $450M+ in­to new Ire­land-based man­u­fac­tur­ing site project and hir­ing spree

As Ireland continues to see more investments and building projects from pharma companies, another contender is looking to place more investment in the Emerald Isle.

According to a report from The Irish Times on Friday, Abbott Laboratories is investing €440 million, or about $451 million, to build a new manufacturing plant in Kilkenny, located in the country’s southeast, to make more of its glucose monitors.

Andy Kidd, Aptinyx CEO

‘The place­bo caught up’: Months af­ter di­a­bet­ic nerve pain fail, Aptinyx sees an­oth­er pain tri­al fall through

In 2019, Aptinyx’s stock cratered after it reported that its lead candidate failed a diabetic nerve pain trial. After some ‘further analysis,’ the biotech re-upped with that same non-opioid pain drug in two more mid-stage studies — another diabetic nerve pain trial and later a fibromyalgia trial.

In April, Aptinyx reported that its second diabetic nerve pain trial also fell through. However, the Illinois-based penny stock biotech had one more shot for its NMDA-modulating drug, dubbed NYX-2925, in fibromyalgia.

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Tony Coles, Cerevel CEO

Cerev­el takes the pub­lic of­fer­ing route, with a twist — rais­ing big mon­ey thanks to ri­val da­ta

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

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Randall Schatzman, Bolt CEO

Bolt throws a wrench in the pipeline, look­ing to con­serve cash

A meltdown in the biotech market is making most execs cautious, including Bolt Biotherapeutics’ CEO, as the company hits the brakes on one preclinical asset and pauses other early-stage work to extend cash reserves by two years.

The pipeline re-org will keep Bolt’s lights on through 2025 so the biotech can focus on its clinical-stage HER2 solid tumor drug candidate, which the company should have early-stage data on and a recommended Phase II dose by year’s end. The biotech also wants to focus on its preclinical asset BDC-3042, an immune-stimulating antibody conjugate (ISAC), for KRAS and TP53 mutated tumors.