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.”

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

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I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

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The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

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While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

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The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

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Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

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Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.