Kristin Fortney, BioAge Labs CEO

An­ti-ag­ing biotech up­start plucks a drug from Am­gen's dis­card pile, piv­ot­ing from heart fail­ure to mus­cle con­di­tions

Back in April 2019, Am­gen qui­et­ly shut down a Phase I tri­al for a drug named AMG 986. There was no safe­ty con­cern; the mol­e­cule just didn’t hit the mark on help­ing the small band of heart fail­ure pa­tients who re­ceived it.

A small biotech, though, be­lieves it would stand a chance in the bur­geon­ing an­ti-ag­ing field.

BioAge Labs has li­censed AMG 986 — now re­named BGE-105 — with plans to par­lay the ex­ist­ing IND in­to a quick Phase I tri­al teas­ing out the phar­ma­co­dy­nam­ic ef­fects and set the stage for mid-stage tests fo­cused on acute mus­cle in­di­ca­tions.

BGE-105 mim­ics the ef­fect of apelin, an en­doge­nous lig­and that boosts the pro­duc­tion of APJ, a re­cep­tor that tends to be down­reg­u­lat­ed as peo­ple grow old­er. By comb­ing through decades of health da­ta from thou­sands of healthy vol­un­teers tracked by their biobank part­ners, the biotech had ze­roed in on the apelin/APJ path­way as one key mol­e­c­u­lar dri­ver of ag­ing, CEO Kris­ten Fort­ney said.

“Our longevi­ty map iden­ti­fies prob­a­bly sev­er­al dozen path­ways that mat­ter for hu­man ag­ing, and we’re choos­ing strate­gi­cal­ly to fo­cus first on the ones that are the most well known,” she added, “where there is as­set avail­able to go im­me­di­ate­ly in­to the clin­ic.”

She imag­ines there are about 10 such pro­grams out there, rep­re­sent­ing a range of mech­a­nis­tic bets, that they can bring in be­fore turn­ing to dis­cov­ery ef­forts. The apelin/APJ one is the third, lin­ing up af­ter two oth­ers tar­get­ing the hy­pox­ia-in­ducible fac­tor and PGD2 DP1.

Along the search for as­sets BioAge con­nect­ed with Cedric Dray at France’s IN­SERM, who had found that in­ject­ing mice with apelin pep­tide en­hanced mus­cle func­tion con­sid­er­ably, but that nat­ur­al mol­e­cule has a short half-life of on­ly 5 min­utes. He helped test the Am­gen mol­e­cule in his mouse mod­els, ce­ment­ing the li­cens­ing deal.

Like most of the an­ti-ag­ing out­fits, Fort­ney ex­pects to start with acute in­di­ca­tions where BioAge can quick­ly gen­er­ate clin­i­cal da­ta and get reg­u­la­to­ry ap­provals — one ex­am­ple is pre­vent­ing mus­cle at­ro­phy for im­mo­bi­lized pa­tients in hos­pi­tals — be­fore mov­ing on to long-term chron­ic use in things like mus­cle re­gen­er­a­tion and frailty.

“Ide­al­ly our drugs would fol­low a de­vel­op­ment path like a statin, where they’re first ap­proved for a nar­row in­di­ca­tion and widen over time” un­til they’re pre­scribed to any­one over a cer­tain age with risk fac­tors, Fort­ney said.

There’s a lot to prove in the young an­ti-ag­ing space, with lofty goals that have been well-doc­u­ment­ed and well-sat­i­rized. Backed by high-pro­file in­vestors like An­dreessen Horowitz and se­r­i­al en­tre­pre­neur Elad Gil of Airbnb fame, BioAge Labs has re­cent­ly brought its to­tal fund­ing to $127 mil­lion and grown the team to 35.

“Frankly very few mech­a­nisms have even been tried in the clin­ic,” she said. “We’re sort of at the very be­gin­ning.”

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.

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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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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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.

Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Phillip Gomez, Siga Technologies CEO

Siga nabs $10.7M from the US gov­ern­ment in deal for its mon­key­pox an­tivi­ral

The US government is all set to buy $10.7 million worth of Siga Technologies’ monkeypox oral antiviral, the company announced Thursday.

Of the total doses, $5.1 million worth of oral antivirals called Tpoxx (tecovirimat) will be delivered this year, with the US Department of Defense having the option of buying the rest at a later point.

The new contract follows an earlier one in which the government had purchased $7.4 million worth of Tpoxx from the company.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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