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.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,100+ biopharma pros reading Endpoints daily — and it's free.

CEO Marshall Fordyce (Vera)

UP­DAT­ED: An am­bi­tious GV-backed gene edit­ing up­start re-emerges as a one-drug kid­ney com­pa­ny

In September 2019, Trucode Gene Repair launched, after two-plus years of early development, with $34 million from GV and Kleiner Perkins and ambitious plans to take on the raft of CRISPR companies with a new form of gene editing.

Those plans, though, would only last a few months. By February, company leadership was already pivoting, filing a trademark and domain name for a new company, called Vera Therapeutics. On Monday, they completed the switch, “launching” with an $80 million Series C round led by Abingworth and plans to focus their efforts on atacicept, a 20-year-old molecule now in development for kidney disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,100+ biopharma pros reading Endpoints daily — and it's free.

Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

Eli Lil­ly tees up dis­cov­ery pact worth more than $1.6B with Merus for T cell-fo­cused bis­pe­cif­ic an­ti­bod­ies

Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,100+ biopharma pros reading Endpoints daily — and it's free.

Sekar Kathiresan (Verve)

'Bluest of blue-chip in­vestors' pump $94M in­to Sekar Kathire­san's quest to bring CRISPR to the heart

Going into his first JP Morgan conference, Sekar Kathiresan had some major news to share. His ambitious cardiovascular-focused startup, Verve Therapeutics, had designated the PCSK9 base editor as its lead program, homed in on severe heterozygous familial hypercholesterolemia as the first indication, and released animal data suggesting the in vivo edits to the monkey livers last up to six months. For a company that had just been in the public for less than a year, “it was wonderful.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,100+ biopharma pros reading Endpoints daily — and it's free.

A car­dio­vas­cu­lar cell ther­a­py play­er grabs $54M for a new be­gin­ning — where next-gen CAR-T ap­proach fea­tures promi­nent­ly

Before the advent of CAR-T, the term cell therapy conjured up very different images. Sure, there were cancer immunotherapies like Dendreon’s Provenge, but more common were the various flavors of stem cell therapies and cell transplantation.

None of that has gone away, even if they’ve been nudged out of the spotlight — and a little biotech has garnered $54 million (£40 million) to show that both the old and new ideas of cell (and gene) therapy can mesh together to form a special breed of platform company.

Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.