Af­ter decades in the big leagues, Mar­tin Mack­ay is go­ing small — and he's de­light­ed

Mar­tin Mack­ay has spent more than three decades in drug dis­cov­ery and de­vel­op­ment, reach­ing top R&D po­si­tions at Pfiz­er, As­traZeneca and Alex­ion, where he en­joyed big bud­gets in pur­suit of block­buster drugs and styl­ish­ly sur­vived a se­ries of big cor­po­rate shake­ups.

Stephen Uden

Now, the Scot­tish re­search ex­ec and some of his ex-Alex­ion col­leagues are go­ing small, in a big way. Mack­ay, for­mer Alex­ion SVP Stephen Uden and for­mer Alex­ion tax chief Jef­frey Fry­er band­ed to­geth­er af­ter the big Lud­wig Hantson purge in 2017 to start Rally­bio at the be­gin­ning of this year. And some mar­quee ven­ture groups are bankrolling their ef­fort with a $37 mil­lion A round.

“I just feel we have some meds left in us,” says Mack­ay. 

Jef­frey Fry­er

The cash will go to build­ing out a small team at Rally­bio, where Mack­ay and his co-founders have been scout­ing rare dis­ease drugs in acad­e­mia and phar­ma, plan­ning to start build­ing a pipeline of drugs with an eye to ex­e­cut­ing a quick piv­ot to reg­u­la­tors.

5AM Ven­tures, Canaan Part­ners, and New Leaf Ven­ture Part­ners led the fi­nanc­ing, with Con­necti­cut In­no­va­tions lend­ing some state sup­port for the Farm­ing­ton, CT-based com­pa­ny.

“It would be eas­i­er to say what we’re not go­ing to do,” Mack­ay tells me. He ticks off the don’t-go-there list: On­col­o­gy, in­fec­tious dis­eases, oph­thal­mol­o­gy and vac­cines. They’re stick­ing with what they know: an­ti­bod­ies, small mol­e­cules and en­gi­neered pro­teins.

There are no ge­o­graph­ic bound­aries to their search. Uden has worked a lengthy stint in Japan. They all have ex­ten­sive Eu­ro­pean ex­pe­ri­ence, Mack­ay’s old stomp­ing grounds be­fore in­vestors drove a purge at As­traZeneca that brought Pas­cal So­ri­ot to the helm. The US is home, but they’re not lim­it­ing them­selves to the big hubs in Boston/Cam­bridge and the Bay Area.

Right now, they have the mon­ey to get to work and some lines on some ear­ly-stage as­sets.

“We’d be look­ing for ear­ly proof-of-con­cept to piv­ot to a reg­u­la­to­ry OK,” says Mack­ay, who’s tak­ing the CEO post at the new com­pa­ny.

Now with a staff of about 8, Mack­ay plans to build a group of 15 sci­en­tists and one busi­ness per­son to keep an eye on the num­bers. He plans to keep it sim­ple — there’s no ad­min­is­tra­tive as­sis­tant to book trav­el — with a bare­bones bud­get.

Mack­ay knows what it’s like to man­age glob­al re­search groups. Now he wants to see just how nim­ble a lit­tle biotech can be.


Im­age: Mar­tin Mack­ay. NO­VO NORDISK

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

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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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Luc Boblet, Egle Therapeutics CEO

A new Treg play­er emerges with $46M and back­ing from Take­da

In recent years, the chorus of biotechs and Big Pharma backers targeting regulatory T cells — also known as “Tregs” — for cancer and autoimmune diseases has only grown louder.

The newest voice is from Egle Therapeutics, which sang out a $46.4 million Series A round on Friday led by LSP and Bpifrance through their InnoBio 2 fund. Takeda’s venture arm also chipped in, about a year after the pharma struck a research pact with the Paris-based upstart.

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René Russo, Xilio CEO (Alicia Petitti for Xilio)

Xilio Ther­a­peu­tics goes pub­lic as bio­phar­ma IPOs rum­ble for­ward in Q4

It’s been a busy fall on Wall Street — and this week, we’ve got another biotech going public after Ventyx yesterday.

Massachusetts solid-tumor biotech Xilio Therapeutics made its public debut today, joining 149 other biotechs that have gone public so far this year.

Back on October 1st, Xilio filed its S-1 with the SEC to make its IPO debut for $100 million — the next step in financing after completing a $95 million Series C back in February.

Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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