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.”

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.

Por­tion of Neil Wood­ford’s re­main­ing in­vest­ments, in­clud­ing Nanopore, sold off for $284 mil­lion

It’s been precisely one year and one day since Neil Woodford froze his once-vaunted fund, and while a global pandemic has recently shielded him from the torrent of headlines, the fallout continues.

Today, the California-based patent licensing firm Acacia Research acquired the fund’s shares for 19 healthcare and biotech companies for $284 million.  Those companies include shares for public and private companies and count some of Woodford’s most prominent bio-bets, such as Theravance Biopharma, Oxford Nanopore and Mereo Biopharma, according to Sky News, which first reported the sale. It won’t include shares for BenevelontAI, the machine learning biotech once valued at $2 billion.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.