French biotech and Pfiz­er find pos­i­tives in da­ta for Ly­me dis­ease vac­cine can­di­date; Al­tim­mune's obe­si­ty drug shows promise in PhI tri­al

French biotech Val­ne­va and part­ner Pfiz­er con­tin­ued to re­veal pos­i­tive re­sults from its Phase II tri­al for its Ly­me dis­ease vac­cine can­di­date.

The Phase II study, which en­rolled 246 healthy adults be­tween 18-65 in the US, met its pri­ma­ry end­point last Oc­to­ber, demon­strat­ing that the can­di­date, known as VLA15, was im­muno­genic across all dose groups test­ed and elicit­ed high an­ti­body re­spons­es one month af­ter the pri­ma­ry vac­ci­na­tion se­ries. Con­tin­ued eval­u­a­tion at Month 18 showed that an­ti­body titers de­clined across all groups, but re­mained above base­line, con­firm­ing the need for a boost­er.

Val­ne­va and Pfiz­er joined forces in a $308 mil­lion deal to com­mer­cial­ize the can­di­date in April 2020. The vac­cine tar­gets the six most com­mon types of Bor­re­lia bac­te­ria that cause Ly­me dis­ease — and Val­ne­va CEO Thomas Lin­gel­bach said he ex­pects to bring the prod­uct to mar­ket in 3-4 years.

VLA15 al­so proved to be safe and well-tol­er­at­ed across all dos­es and age groups test­ed in the tri­al. No re­lat­ed se­ri­ous ad­verse events were ob­served in any treat­ment group, the com­pa­nies said.

“We are ex­cit­ed by these ad­di­tion­al Phase 2 re­sults, which we be­lieve take us a step clos­er to mak­ing a ma­jor con­tri­bu­tion against this se­vere dis­ease, sub­ject to reg­u­la­to­ry ap­proval,” said Val­ne­va CMO Juan Car­los Jaramil­lo in a state­ment.

Al­tim­mune finds ear­ly suc­cess from Phase I tri­al of obe­si­ty drug can­di­date 

Bio­phar­ma Al­tim­mune re­leased promis­ing re­sults from a 12-week, Phase I tri­al for its obe­si­ty drug can­di­date, find­ing sig­nif­i­cant weight loss among some par­tic­i­pants, with high­er rates of nau­sea and vom­it­ing among those on the high­er dos­es.

The Aus­tralian tri­al of the GLP-1/glucagon dual re­cep­tor ag­o­nist pemvidu­tide, for­mer­ly known as ALT-801, in 34 sub­jects showed an av­er­age weight loss of be­tween 5% and 10% at dif­fer­ent dos­es, de­pend­ing on the dose. Those on place­bo av­er­aged 1.6% weight loss, ac­cord­ing to an Al­tim­mune state­ment.

“Giv­en that these weight loss da­ta were ob­tained with­out di­et or be­hav­ioral mod­i­fi­ca­tions, we are ex­cit­ed to see weight loss reach its full po­ten­tial dur­ing the planned 48-week Phase II obe­si­ty tri­al next year,” said Al­tim­mune CMO Scott Har­ris in a state­ment.

Al­tim­mune said back in Ju­ly that the com­pa­ny planned to piv­ot fo­cus back to the obe­si­ty drug can­di­date af­ter dis­ap­point­ing clin­i­cal re­sults with its Covid-19 vac­cine can­di­date. ALT’s stock price was -20% pre-mar­ket as of this morn­ing.

Hill­stream files $17m IPO

Hill­stream Bio­Phar­ma filed an IPO on Mon­day with the SEC to raise up to $17 mil­lion.

The Bridge­wa­ter, NJ bio­phar­ma plans to raise $17 mil­lion by of­fer­ing 3 mil­lion shares at a price range of be­tween $5 and $6. At the mid­point of the pro­posed range, Hill­stream Bio­Phar­ma would com­mand a ful­ly di­lut­ed mar­ket val­ue of $58 mil­lion, ac­cord­ing to a Hill­stream state­ment. Hill­stream plans to list on the Nas­daq un­der the sym­bol $HILS.

The com­pa­ny has been fo­cused on fer­rop­to­sis, a type of pro­grammed cell death be­ing stud­ied for treat­ment re­sis­tant can­cers. The com­pa­ny’s most ad­vanced can­di­date is HSB-1216, an iron me­di­at­ed cell death, or IM­CD in­duc­er for sol­id tu­mors. Hill­stream aims to sub­mit an IND and start a clin­i­cal study with the drug next year.

FDA ac­cepts PD-1 drug Lib­tayo for pri­or­i­ty re­view against cer­vi­cal can­cer

Re­gen­eron an­nounced this morn­ing that the FDA has ac­cept­ed PD-1 in­hibitor Lib­tayo for pri­or­i­ty re­view to po­ten­tial­ly treat dif­fer­ent types of cer­vi­cal can­cer.

The tar­get ac­tion date for the FDA de­ci­sion is Jan. 30, 2022. The sBLA is be­ing re­viewed un­der the FDA’s Pro­ject Or­bis ini­tia­tive, which will al­low for con­cur­rent re­views by oth­er health au­thor­i­ties in Aus­tralia, Brazil, Cana­da and Switzer­land. Re­gen­eron is al­so plan­ning ad­di­tion­al glob­al reg­u­la­to­ry sub­mis­sions, in­clud­ing in the EU, by the end of the year.

Lib­tayo was joint­ly de­vel­oped by Re­gen­eron and Sanofi and first ap­proved in 2018 for lo­cal­ly ad­vanced and metasta­t­ic cu­ta­neous squa­mous cell car­ci­no­ma — then ap­proved in 2021 for NSCLC and basal cell car­ci­no­ma.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

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