Months af­ter $10M Se­ries A, rare dis­ease AI up­start Healx hauls in $56M in fresh fund­ing

In 2010, Nick Sireau quit his job to fo­cus sole­ly on the pa­tient group he had set up to help his sons di­ag­nosed with a rare ge­net­ic dis­ease called alka­p­tonuria (AKU). Re­searchers had found a fea­si­ble treat­ment from an un­like­ly source: a weed­killer, but it was be­ing used to help in­fants with a dif­fer­ent dis­or­der. Sireau would then con­front the per­ils of the tra­di­tion­al tri­al-and-er­ror drug dis­cov­ery process that de­mands time and mon­ey.

Tim Guil­liams

In 2014, two sci­en­tists met Sireau, who had been fierce­ly lob­by­ing and fundrais­ing to get UK au­thor­i­ties to tri­al the com­pound in pa­tients with AKU. The en­counter sparked the two re­searchers — the British sci­en­tist be­hind Vi­a­gra, David Brown, and a bio­engi­neer from Cam­bridge, Tim Guil­liams — to work on mar­ry­ing the con­cept of drug re­pur­pos­ing with AI and ma­chine learn­ing, par­tic­u­lar­ly for rare con­di­tions. Soon, their com­pa­ny — Healx — was born.

This Ju­ly, the Cam­bridge, UK-based com­pa­ny raised $10 mil­lion in a Se­ries A round of fund­ing. On Wednes­day, Healx un­veiled a fresh $56 mil­lion in­jec­tion — as it preps for a Phase IIa clin­i­cal tri­al ex­pect­ed to ini­ti­ate in the first quar­ter of 2020 — in pa­tients with Frag­ile X syn­drome, a con­di­tion that spawns de­vel­op­men­tal prob­lems and is con­sid­ered the lead­ing ge­net­ic cause of autism.

Nick Sireau

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The tri­al will eval­u­ate the safe­ty and ef­fi­ca­cy of a com­bi­na­tion ther­a­py, Guil­liams told End­points News, de­clin­ing to pro­vide fur­ther de­tail.

“If you look at com­bi­na­tions of two or three drugs, you have about 13 bil­lion pos­si­bil­i­ties per dis­ease — so how do you se­lect the top 10, the top 20? And we don’t go be­yond that,” Guil­liams said. Healx’s AI plat­form claims to short­en the dis­cov­ery-to-clin­ic time­line to as lit­tle as 24 months.

David Brown

Healx’s ap­proach does not lean on an ini­tial hy­poth­e­sis. “We let the al­go­rithm de­cide which dis­eases we’re go­ing to work on, which drugs are be­ing matched. And then we work out a mech­a­nism or hy­poth­e­sis af­ter­wards,” he said.

So far, Healx has launched 10 pre­clin­i­cal pro­grams across a pletho­ra of in­di­ca­tions — of which four (in­clud­ing the Frag­ile X pro­gram) have reaped re­sults so far, Guil­liams said, not­ing that each of these read­outs has been pos­i­tive. With this Se­ries B, the plan is to launch an­oth­er 40 pre­clin­i­cal pro­grams, he added.

If they are suc­cess­ful — with any of these shots on goal — the plan is to make sure pric­ing is not in the typ­i­cal or­phan drug price range which hov­ers around $240,000 per pa­tient per year, Guil­liams said.

Re­pur­pos­ing drugs on pur­pose (or by ac­ci­dent) has yield­ed some suc­cess — that the process in­volves large­ly de-risked com­pounds, low­er de­vel­op­men­tal costs, and briefer time­lines doesn’t hurt ei­ther.

Vi­a­gra is, of course, the most heav­i­ly cit­ed ex­am­ple. The drug, known chem­i­cal­ly as silde­nafil, was orig­i­nal­ly be­ing test­ed as a treat­ment for coro­nary hy­per­ten­sion — but a pesky side ef­fect felt by pa­tients in tri­als led to its even­tu­al ap­proval as an erec­tile dys­func­tion drug.

Then there’s the seda­tive thalido­mide — which gained no­to­ri­ety af­ter its link to se­vere skele­tal birth de­fects trig­gered its with­draw­al in 1957. How­ev­er, years lat­er it was deemed ef­fec­tive as a can­cer treat­ment, even breed­ing the de­vel­op­ment and ap­proval of even more suc­cess­ful de­riv­a­tives, such as Cel­gene’s block­buster Revlim­id.

Mean­while, Mer­ck’s Vioxx — which was un­cer­e­mo­ni­ous­ly tak­en off shelves af­ter its link to dou­bling pa­tients’ risk of heart at­tack and stroke emerged — could resur­face as a gener­ic treat­ment for a side ef­fect ex­pe­ri­enced by he­mo­phil­ia pa­tients. The gener­ic ver­sion is be­ing prepped for a piv­otal tri­al slat­ed to be­gin next year.

But akin to tra­di­tion­al drug de­vel­op­ment, drug re­pur­pos­ing has al­so seen its share of set­backs. Two ex­am­ples of late-stage fail­ures in­clude the bid to use the an­ti­his­t­a­mine, la­trepir­dine, as a treat­ment for Hunt­ing­ton’s dis­ease, as well as the pur­suit of re­pur­pos­ing the an­tibi­ot­ic, cef­tri­ax­one, as a med­i­cine for ALS.

Healx’s Se­ries B was led by Eu­ro­pean VC firm Atom­i­co and joined by Glob­al Brain and btov Part­ners. All pre­vi­ous in­vestors, in­clud­ing Balder­ton Cap­i­tal, Amadeus Cap­i­tal Part­ners and Jonathan Mil­ner, al­so par­tic­i­pat­ed in the round.

Healx did not ini­tial­ly plan to raise more mon­ey this year, but in­ter­est in their ap­proach bal­looned giv­en their progress, Guil­liams said. “I think that’s a re­al­ly good sign for the in­vest­ment land­scape in the UK and Eu­rope be­cause for Eu­rope it’s a pret­ty large B round…com­pared to the US.”

“And I’m won­der­ing if part of that is let’s make sure we in­vest be­fore Brex­it kicks in.”

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Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

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Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

Mod­er­na los­es lat­est bat­tle in key vac­cine de­liv­ery patent fight as fed­er­al ap­peal falls flat

The US Court of Appeals for the Federal Circuit on Wednesday rejected Moderna’s attempt to overturn key patents related to the delivery vehicle for its Covid-19 vaccine after the biotech sought to preempt a potentially risky infringement lawsuit.

For years, Moderna has been battling a tiny Pennsylvania biotech known as Arbutus over patents for a technology required to deliver its mRNA drugs and vaccines, known as lipid nanoparticles or LNP. Moderna is concerned there’s a substantial risk that Arbutus will assert the ’069 patent in an infringement suit targeting Moderna’s Covid-19 vaccine, particularly as Arbutus has boasted of its patent protection and refused to grant a covenant not to sue Moderna.

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Lan Huang, BeyondSpring CEO

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The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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What's fair? New ICER re­port shows pay­ers gen­er­al­ly en­sur­ing fair ac­cess to drugs

The nonprofit Institute for Clinical and Economic Review on Wednesday released a new report highlighting the ways in which payers are generally ensuring fair access to prescription drugs, even when based on a set of criteria set by the nonprofit.

While noting the lack of transparency hindered the report’s results, ICER said that the “great majority” of payer policies in the formularies evaluated are structured in a way to support many key elements of how ICER defines “fair access.”