Re­gen­eron/Sanofi PD-1 tri­al halt­ed for sig­nif­i­cant ef­fi­ca­cy; Avadel soars on pos­i­tive nar­colep­sy da­ta

→ It’s a good news, bad news day for Sanofi and Re­gen­eron. On the same morn­ing their joint Kevzara tri­al pro­duced dis­ap­point­ing re­sults for Covid-19, the biotech-phar­ma part­ners an­nounced that a Phase III tri­al test­ing their im­munother­a­py Lib­tayo on non-small cell lung can­cer had been stopped ear­ly af­ter hav­ing shown clear ben­e­fit in im­prov­ing over­all sur­vival. The PD-1 in­hibitor re­duced the risk of death by 32% in first-line pa­tients.

→ Irish drug de­vel­op­er Avadel Phar­ma­ceu­ti­cals‘ shares $AVDL shot up near­ly 33% to $13.06 in Mon­day pre­mar­ket trad­ing, af­ter the com­pa­ny un­veiled pos­i­tive late-stage da­ta on its ex­per­i­men­tal drug, FT218, un­der eval­u­a­tion as a treat­ment for ex­ces­sive day­time sleepi­ness and cat­a­plexy in pa­tients with nar­colep­sy. The REST-ON tri­al met its three co-pri­ma­ry ef­fi­ca­cy end­points at all three dos­es, in­duc­ing sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments on mea­sures of wake­ful­ness and mean week­ly cat­a­plexy (sud­den mus­cu­lar weak­ness trig­gered by strong emo­tions such as laugh­ter, anger and sur­prise) at­tacks.

Akin to Jazz Phar­ma­ceu­ti­cals‘ cash cow nar­colep­sy ther­a­py Xyrem, FT218 is al­so a for­mu­la­tion of sodi­um oxy­bate. “(P)os­i­tive FT218 da­ta de­risk ap­proval from a clin­i­cal per­spec­tive, and al­so, with the caveats of cross-tri­al com­par­isons, sug­gest ef­fi­ca­cy on par with Jazz’s Xyrem,” Stifel’s Paul Mat­teis wrote in a note.

Neu­ro­crine Bio­sciences, which last year joined forces with Voy­ager Ther­a­peu­tics to work on a range of gene ther­a­pies, has scored FDA ap­proval for On­gen­tys, an add-on ther­a­py de­signed to sup­ple­ment stan­dard Parkin­son’s ther­a­py lev­odopa/car­bidopa.

As Parkin­son’s dis­ease pro­gress­es, first-line treat­ments such as lev­odopa be­gin to lose ef­fec­tive­ness and the ben­e­fi­cial ef­fects of the drug be­gin to wear off more quick­ly, caus­ing more fre­quent and of­ten de­bil­i­tat­ing mo­tor fluc­tu­a­tions in pa­tients — On­gen­tys has shown to sig­nif­i­cant­ly re­duce that “off” time, cul­mi­nat­ing in bet­ter and more con­sis­tent mo­tor symp­tom con­trol. The drug is set to launch lat­er in 2020, the com­pa­ny said.

Take­da‘s ex­per­i­men­tal tar­get­ed can­cer drug, mobo­cer­tinib, has se­cured the FDA’s break­through ther­a­py sta­tus for pa­tients with a form of non-small cell lung can­cer with epi­der­mal growth fac­tor re­cep­tor (EGFR) ex­on 20 in­ser­tion mu­ta­tions whose dis­ease has pro­gressed on or af­ter plat­inum-based chemother­a­py. “Al­though most EGFR mu­ta­tions can be tar­get­ed by cur­rent­ly avail­able TKIs (Ty­ro­sine ki­nase in­hibitors), peo­ple with ex­on 20 in­ser­tion mu­ta­tions of­ten suf­fer and feel for­got­ten since avail­able EGFR in­hibitors don’t work well in their can­cer,” said Jill Feld­man, lung can­cer pa­tient and co-founder of EGFR Re­sisters, in a state­ment.

→ First, Har­poon got a cool $50 mil­lion for al­low­ing Ab­b­Vie the op­tion to li­cense its ex­per­i­men­tal drug, HPN217. Now, as the com­pa­ny kicks off a Phase I/II tri­al test­ing the ther­a­py in re­lapsed, re­frac­to­ry mul­ti­ple myelo­ma pa­tients, the US drug­mak­er is fork­ing over an­oth­er $50 mil­lion.

→ Chi­na biotech Green Val­ley grabbed a sig­nif­i­cant amount of at­ten­tion re­cent­ly when it bagged an ap­proval in its home coun­try for an Alzheimer’s drug. Now the FDA is al­low­ing their re­searchers to con­duct a US test that will de­cide its fate out­side of Chi­na. Give this one a long lead time, though. The com­pa­ny doesn’t ex­pect to file be­fore 2025, when all the da­ta are in.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

ARCH-backed SciNeu­ro kicks off search for CNS au­toan­ti­bod­ies with new deal; Mer­ck + Gilead an­nounce PhII tri­al for HIV com­bo

From the very beginning at SciNeuro, CEO Min Li has envisioned a mix of licensing deals and scientific efforts to replicate the breakout success of China’s oncology companies in neuroscience.

The GlaxoSmithKline vet has now inked a deal that somewhat straddles the line between the two strategies.

Teaming up with Mabylon out of Zurich, SciNeuro is now looking to test the hypothesis that the human immune system can play a role in fighting neurodegenerative diseases by discovering and developing human autoantibodies against neurological “targets of mutual interests.” The new partners offered TAR DNA binding protein-43 (TDP-43) and apolipoprotein E (APOE), which are linked to ALS and Alzheimer’s, as examples.

Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.