Vipin Garg, Altimmune CEO

Al­tim­mune aban­dons ship on in­tranasal Covid-19 vac­cine, re­fo­cus­ing on liv­er, obe­si­ty pro­grams af­ter tri­al flop

Among the al­so-rans in the Covid-19 vac­cine race, small biotech Al­tim­mune hoped to ride a nov­el in­tranasal for­mu­la­tion for its own can­di­date to mar­ket but ran in­to an FDA clin­i­cal hold along the way. Now, af­ter the vac­cine turned out a dud in an ear­ly-stage test, Al­tim­mune is bail­ing out and re­fo­cus­ing around its oth­er pro­grams.

Al­tim­mune’s sin­gle-dose vac­cine, dubbed Ad­COVID, didn’t spur an ad­e­quate im­mune re­sponse in a Phase I test in 80 healthy vol­un­teers, the biotech said Tues­day. Mean­while, the vac­cine was well tol­er­at­ed, but the lack of re­sults was good enough for Al­tim­mune to walk away.

Al­tim­mune CEO Vipin Garg called the re­sults “dis­ap­point­ing” in a state­ment, par­tic­u­lar­ly af­ter the biotech thought it saw promise in pre­clin­i­cal stud­ies. Shares of the biotech $ALT were trad­ing down near­ly 33% af­ter the bell at around $10.

Al­so on Tues­day, Al­tim­mune re­vealed it would hit the pause but­ton on its less­er-known im­munomod­u­la­to­ry can­di­date for Covid-19, dubbed T-COVID, af­ter run­ning in­to dif­fi­cul­ty en­rolling high-risk Covid-19 pa­tients above the age of 65. The com­pa­ny at­trib­uted that chal­lenge to the suc­cess­ful roll­out of Covid-19 vac­cines in the US and said they would end de­vel­op­ment in the high-risk co­hort while feel­ing out a po­ten­tial path in low-risk pa­tients.

Ad­COVID’s road to the clin­ic was de­layed back in De­cem­ber af­ter the FDA put a halt on the com­pa­ny’s IND ap­pli­ca­tion af­ter cit­ing is­sues with the vac­cine’s tri­al pro­to­col and CMC is­sues. Af­ter agree­ing to the FDA’s de­mands, Al­tim­mune opened en­roll­ment in Phase I in Feb­ru­ary, giv­ing pa­tients ei­ther one or two dos­es of the vac­cine at three sep­a­rate dose lev­els.

The biotech said its vac­cine did spur some an­ti­body re­sponse but not enough to keep pace with the vac­cines al­ready ap­proved for emer­gency use.

Mean­while, Al­tim­mune said it plans to piv­ot fo­cus back to ALT-801, a can­di­date for obe­si­ty, and Hep­T­cell, a pep­tide-based ther­a­py for obe­si­ty and liv­er dis­eases.

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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#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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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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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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#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: Iver­ic Bio, un­de­terred by loom­ing PDU­FA for com­peti­tor, touts sub­group da­ta on GA drug

CHICAGO — While its competitor is on the cusp of likely securing the first FDA nod, Iveric Bio is trudging ahead with its potential treatment for geographic atrophy, an advanced form of AMD, and has new data to support its upcoming NDA filing.

The biotech said its drug was more favorable than sham across all subgroups in the second Phase III study of the investigational complement C5 protein inhibitor, performing similarly to a previous, smaller late-stage trial.

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#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

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