Greg Miller, Transition Bio CEO

The tar­get is the sys­tem: Con­den­sates biotech as­pires to ‘rewrite the rule­book’ on drug dis­cov­ery

An­oth­er biotech has burst in­to the buzzy but still bud­ding field of bio­mol­e­c­u­lar con­den­sates with $50 mil­lion in Se­ries A fund­ing.

Tran­si­tion Bio, based in both Cam­bridge, UK, and Cam­bridge, MA, is look­ing to bring con­den­sate drug dis­cov­ery, a field that orig­i­nat­ed in neu­ro­science, to can­cer. Co-found­ed by two bio­physics pro­fes­sors — David Weitz of Har­vard and Tuo­mas Knowles of the Uni­ver­si­ty of Cam­bridge — the 18-month-old biotech is build­ing a ma­chine learn­ing plat­form that maps out con­den­sates in what Knowles de­scribed as a “phase di­a­gram.”

First ob­served in worm cells in 2009, con­den­sates are tran­sient droplets that help or­ga­nize the in­sides of cells, “bring­ing to­geth­er what needs to be to­geth­er,” Knowles said. But un­like or­ganelles, which sep­a­rate out a cell’s con­tents with phys­i­cal bar­ri­ers, con­den­sates act like liq­uids, con­dens­ing (hence the name) and dis­solv­ing to bring pro­teins to­geth­er or set them apart.

Tuo­mas Knowles

“Typ­i­cal­ly a healthy con­den­sate would be formed in a high­ly re­versible man­ner, such that when it’s no longer re­quired it can dis­solve and not clog up any of the oth­er cel­lu­lar ma­chin­ery,” Knowles said.

How­ev­er, dis­ease-re­lat­ed con­den­sates can mal­func­tion in a num­ber of ways. In some cas­es, they may gel to­geth­er so they’re no longer liq­uid and dis­solv­able, Knowles said. In oth­ers, con­den­sates may bring to­geth­er the wrong com­bi­na­tion of mol­e­cules or ap­pear where they don’t in healthy cells.

Us­ing its plat­form, Tran­si­tion Bio plans on map­ping out the many dif­fer­ent con­di­tions that lead to con­den­sate for­ma­tion and func­tion in cells. By do­ing so, the biotech hopes it can use ma­chine learn­ing to fig­ure out how to reg­u­late con­den­sates.

“Ar­guably, much of what we know about what makes a drug-like mol­e­cule is based on drug­ging sin­gle pro­teins,” Knowles said, “so those rules are like­ly to look very dif­fer­ent once we start to drug these very high­ly mul­ti­mer­ic and dy­nam­ic sys­tems.

“So we re­al­ly have to — in some sense — rewrite and re­dis­cov­er the rule book,” Knowles added.

There are a hand­ful of oth­er con­den­sate biotechs out there, in­clud­ing Dew­point Ther­a­peu­tics and Nereid, found­ed by the bio­physi­cists that penned the ini­tial worm study. In Feb­ru­ary, Dew­point, which has part­ner­ships with Bay­er and Mer­ck, raised an­oth­er $150 mil­lion hop­ing to get in­to the clin­ic by the end of next year.

For Tran­si­tion Bio, the next step will be to gen­er­ate in vi­vo val­i­da­tion for their con­den­sate sys­tem tar­gets, CEO Greg Miller said.

The Se­ries A round was led by North­pond Ven­tures and in­clud­ed Tai­ho Ven­tures, Bris­tol My­ers Squibb, Mag­net­ic Ven­tures, and Life­force Cap­i­tal, which led Tran­si­tion Bio’s seed round.

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.

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

BREAK­ING: 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.

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.

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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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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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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