Rallybio founders (left to right): Jeff Fryer, Martin Mackay and Steve Uden

Mar­tin Mack­ay’s rare dis­ease start­up gets $145 mil­lion as it en­ters the clin­ic

Two years ago, Mar­tin Mack­ay took a cou­ple of fel­low ex­iles from Alex­ion’s 2017 culling and, af­ter 3 decades in Big Phar­ma and big biotech, struck out on his own, with a vague vi­sion that was as small as it was broad. They called it Rally­bio and it was go­ing to fo­cus on rare dis­eases — they just didn’t say which of the thou­sands of those rare dis­eases that would be.

To­day, they did, dis­clos­ing the first of what they say will even­tu­al­ly be a list of pro­grams. And the trio — which al­so in­cludes for­mer Alex­ion re­search head Stephen Uden and Alex­ion tax chief Jeff Fry­er — raised $145 mil­lion in Se­ries B fund­ing to take in­to the clin­ic, grab­bing cap­i­tal from the apt­ly named Piv­otal bioVen­ture Part­ners and 10 oth­er in­vest­ment firms.

First up is FNAIT, or fe­tal and new-neona­tal al­loim­mune throm­bo­cy­tope­nia, a rare con­di­tion in which a fe­tus has anti­gen on platelet cells that a moth­er lacks. The moth­er, rec­og­niz­ing those anti­gens as for­eign, can mount an im­mune at­tack, de­vel­op­ing an­ti­bod­ies that tar­get the fe­tus’ platelets, lead­ing to bleed­ing, in­clud­ing some­times in the brain. The dis­ease is gen­er­al­ly treat­ed with close mon­i­tor­ing of the ba­by af­ter birth, but Rally­bio wants to pre­vent it, giv­ing moth­ers an an­ti­body that will turn down the im­mune re­sponse ear­ly in the preg­nan­cy.

“The trick will be iden­ti­fy­ing moth­ers at risk,” Uden told End­points News, adding they can do that with a sim­ple test al­ready in use. “It would be a case of re­pur­pos­ing.”

Should the ther­a­py work, the goal would be to have the screen­ing be­come part of the rou­tine rare dis­ease tests women re­ceive dur­ing preg­nan­cy.

The goal “is not to use this to treat the dis­ease but to fun­da­men­tal­ly erad­i­cate it,” Uden said.

The tri­al will be­gin in the sec­ond half of this year, at a sin­gle site in Ger­many, one of the coun­tries that has done sig­nif­i­cant epi­demi­o­log­i­cal work on the dis­ease, with talks un­der­way for how to screen a min­i­mum num­ber of moth­ers.

”It’s not like we’re a big com­pa­ny run­ning hun­dreds of tri­als — they have un­doubt­ed­ly been hit,” Mack­ay said. “The fact that we’re fo­cused on a small num­ber will help us steer our way through.”

When Rally­bio launched in 2018, the founders had ideas for the dis­ease they want­ed to fo­cus on, but the search picked up af­ter the Se­ries A in April. They es­tab­lished a net­work of es­sen­tial­ly drugs scouts in coun­tries from Is­rael to Japan, hop­ing to find se­ri­ous ill­ness­es whose bi­ol­o­gy was un­der­stood, a so­lu­tion that seemed fea­si­ble but which sim­ply hadn’t got­ten suf­fi­cient at­ten­tion from peo­ple with mon­ey and de­vel­op­ment ex­pe­ri­ence.

Late that fall, Uden met with biotechs from Nordic coun­tries on a win­try day in New York City, in­clud­ing the CEO of Pro­phylix, a biotech fo­cused on FNAIT. Pro­phylix’s ex­pe­ri­ence was large­ly aca­d­e­m­ic, with few re­sources or his­to­ry in busi­ness, but the dis­ease and the treat­ment were fair­ly straight­for­ward. A sim­i­lar ap­proach has long been in rhe­sus dis­ease, a sim­i­lar con­di­tion that matched red blood cells in­stead of platelets. It matched Rally­bio’s cri­te­ria for a good can­di­date.

“How bad is the dis­ease, how good is the bi­ol­o­gy, and can we come in with an in­ter­ven­tion that will make a dif­fer­ence?“ Mack­ay said, de­scrib­ing their cri­te­ria.

Rally­bio ac­quired Pro­phylix and signed deals with two oth­ers, but the goal is to ul­ti­mate­ly have 4 to 8 pro­grams at dif­fer­ent stages of de­vel­op­ment. The busi­ness mod­el de­pends on it, said Mack­ay. If you can bring in ex­per­i­men­tal drugs for sev­er­al of these dis­eases, you can treat more pa­tients, ex­pand your rev­enue and de­risk the plat­form.

“We want to build a sus­tain­able com­pa­ny, we see that as the way to make the most mean­ing­ful dif­fer­ence in these rare and se­vere dis­eases,” he said. “To be a sus­tain­able com­pa­ny you need a port­fo­lio.”

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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