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.”

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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UP­DAT­ED: Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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FDA puts pe­di­atric aGVHD drug on pri­or­i­ty re­view lane — will they go vir­tu­al with the ad­comm?

Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.

With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.

Covid 19 roundup: Trump push­es his new fa­vorite, untest­ed drug; CRISPR out­lines crip­pling im­pact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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ITeos nabs $125M as they prep Keytru­da com­bi­na­tion tri­al — if Covid-19 will let them

For iTeos, it turned out, $75 million could only last so long.

Two years after announcing their eye-catching Series B raise, the Belgian biotech is back with an even larger Series B-2: $125 million.

The now $200 million financing illustrates the vast capital available for those with promising new immuno-oncology compounds, particularly those that might be used in combination with existing therapies. In December, iTeos announced a collaboration with Merck to test its lead compound with Keytruda this year. The proceeds will push forward that trial and help fund the ongoing Phase I/II trials for that compound, EOS-850, and a second one, EOS-448.

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Flex­ion se­cures Chi­na deal for os­teo­poro­sis drug; Strug­gling to find a buy­er, Ako­rn throws in the tow­el

→ Flexion may be hitting the brakes on clinical trials, including one for its osteoporosis Zilretta, but that’s not stopping the biotech from plotting regulatory action in China. Hong Kong Tainuo has committed $10 million upfront to seize the development and commercialization rights to Zilretta, with plans to apply for a clinical trial in China by the end of the year. Flexion, which said it has 10 months of finished goods in the US and 12 months of active pharmaceutical ingredient available, will supply all products to the Chinese partner.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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