Dan Siegwart (UT Southwestern)

Cou­pling mR­NA with tR­NA and new de­liv­ery tech, MPM and Or­biMed help birth new $80M biotech in­to a slowed down world

Part­ners at MPM and Or­biMed were hop­ing David Lock­hart could help them with due dili­gence when they brought him and his team at Tran­scrip­Tx in to look at a tiny up­start spun out of the Uni­ver­si­ty of Texas South­west­ern Med­ical Cen­ter in Dal­las. Dubbed Re­Code Ther­a­peu­tics, it was de­vel­op­ing a lipid nanopar­ti­cle (LNP) de­liv­ery plat­form for var­i­ous ge­net­ic med­i­cines, with a lead pro­gram in cys­tic fi­bro­sis backed by the CF Foun­da­tion.

But Lock­hart, the for­mer CSO of Am­i­cus Ther­a­peu­tics and a one-time biotech en­tre­pre­neur, saw an im­me­di­ate match with the mR­NA and res­pi­ra­to­ry dis­ease work that Tran­scrip­Tx was do­ing. Since his com­pa­ny was al­so look­ing to raise mon­ey, wouldn’t it make sense to merge the two?

So in­stead of two Se­ries B rounds, MPM and Or­biMed have now put their mon­ey be­hind a brand new biotech that’s tak­ing the Re­Code name and $80 mil­lion in Se­ries A cash to hus­tle to­ward the clin­ic and get se­ri­ous about BD — al­beit at a slight­ly slow­er pace amid a pan­dem­ic.

“We closed the merg­er and the fi­nanc­ing on March 6,” Lock­hart told End­points News. “Even though in some ways that wasn’t very long ago, the world was kind of in a dif­fer­ent place even three weeks ago.”

Colt Ven­tures co-led the round with Or­biMed, with par­tic­i­pa­tion from Vi­da Ven­tures, Hunt Tech­nol­o­gy Ven­tures and Os­age Uni­ver­si­ty Part­ners.

The aca­d­e­m­ic labs at UT South­west­ern, where founders Dan Sieg­wart and Philip Thomas are based, have been shut down. With most of the staff work­ing from home, the CEO of­ten finds him­self work­ing in an emp­ty of­fice in Men­lo Park by him­self. Re­Code doesn’t plan to pull the trig­ger on any new hires in the next few weeks.

What they are do­ing, though, is lay­ing the ground­work so that they have every­thing lined up to go at full speed once they can, from pre­clin­i­cal ex­per­i­ments to job can­di­dates. The sci­en­tists who are tend­ing to cru­cial cell lines are al­so still at work while keep­ing their dis­tance, which is rel­a­tive­ly easy with a team of 12 in Cal­i­for­nia and just 3 in Texas.

The orig­i­nal lead pro­gram at Tran­scrip­Tx was de­signed to treat the rare ge­net­ic dis­or­der pri­ma­ry cil­iary dysk­i­ne­sia (PCD) us­ing an mR­NA drug de­liv­ered by an LNP li­censed ear­li­er. While Re­Code is mov­ing for­ward with it, the team will al­so test oth­er can­di­dates us­ing the pro­pri­etary plat­form in par­al­lel. If the re­sults pan out, Re­Code will like­ly swap it out, Lock­hart said.

Not on­ly is the class of LNPs in­vent­ed by Sieg­wart — who stud­ied un­der MIT whiz Robert Langer — eas­i­er to make and ver­sa­tile in the pay­load they can car­ry, they can al­so be de­liv­ered sys­tem­at­i­cal­ly then dis­trib­uted to dif­fer­ent tis­sue types.

Philip Thomas

There are no ap­proved treat­ments for PCD, and as with CF be­fore Ver­tex’s ther­a­pies came along, pa­tients can die of res­pi­ra­to­ry fail­ure in their 30s or 40s even on the best sup­port­ive care. And sev­er­al ge­net­ic mu­ta­tions are known to cause the dis­ease.

“PCD alone is a pipeline that will be big­ger than the Gen­zyme pipeline that made Gen­zyme what it was,” Lock­hart said, cit­ing the rare dis­ease gi­ant that Am­i­cus was of­ten com­pared to.

In CF, Ver­tex has made dra­mat­ic break­throughs with its ar­se­nal of drugs tar­get­ing mu­tant CFTR pro­teins, cov­er­ing 90% of pa­tients. Re­Code’s goal is to catch the oth­er 10%: those with a non­sense mu­ta­tion that pre­vents a full length pro­tein from be­ing pro­duced in the first place. With the LNP tech, its ther­a­py ush­ers in a piece of trans­fer RNA — a bridge be­tween mes­sen­ger RNA and pro­tein syn­the­sis — to the pre­ma­ture stop codon and al­ters the cell ma­chin­ery, coax­ing it to make a func­tion­al ver­sion of CFTR.

PTC Ther­a­peu­tics has tak­en a sim­i­lar read-through ap­proach with its small mol­e­cule drug, but while ataluren has demon­strat­ed (con­tro­ver­sial) ef­fect in Duchenne mus­cu­lar dy­s­tro­phy, it failed in CF.

“They’re ac­tu­al­ly sort of sis­ter dis­eases in some ways,” he added. “The same doc­tors that treat PCD treat peo­ple with CF.”

Be­yond these ini­tial pro­grams, for which clin­i­cal tri­als are still ex­pect­ed in 2021, there’s plen­ty of room for li­cens­ing and col­lab­o­ra­tions. It was a key top­ic for the new board of di­rec­tors, now com­pris­ing Or­biMed’s Pe­ter Thomp­son, MPM’s Ed Hur­witz, Vi­da Ven­tures’ He­len Kim, R.A. Ses­sion from Bridge­Bio and Michael Tor­res, Re­Code founder and new VP of R&D.

“We had our first board meet­ing with the new board just on Fri­day,” Lock­hart said. “Every­one was in a room by them­selves talk­ing in­to a phone.”

UP­DAT­ED: A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

How could a small, largely unknown biotech that emerged from stealth mode just months ago with early-stage cancer programs jump onto Wall Street in the middle of a Category 6 financial hurricane and sail through with a $165 million IPO?

And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

The biotech is a company called Zentalis. The crew there nabbed an $85 million crossover round late last year — notably waiting 5 years before waving the numbers around to attract attention, according to my read of a FierceBiotech story. Perceptive joined in, but the syndicate was not in general the kind of marquee affair that gets tongues wagging.

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Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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Lil­ly Asia Ven­tures co-leads $100M+ round for Chi­nese biotech and its late-stage lu­pus drug

Can a Chinese biotech deliver the first new lupus drug in decades? A high-profile group of VCs are betting on it.

Lilly Asia Ventures and Lake Bleu Capital are the co-headliners for RemeGen’s latest raise, which brought in more than $100 million. Hudson Bay Capital and Vivo Capital — which, like LAV, also invested in a pre-IPO round for Legend Biotech unveiled today — chimed in, as did Janchor Partners and OrbiMed.

GSK's asth­ma bi­o­log­ic Nu­cala is one step clos­er to ap­proval in key chron­ic rhi­nos­i­nusi­tis pop­u­la­tion

Months after GSK’s Nucala cleared in a pivotal rare blood disorder study, the asthma biologic has scored in a late-stage trial in chronic rhinosinusitis patients with nasal polyps.

The British drugmaker on Friday disclosed data from the SYNAPSE study, which tested Nucala (also known as mepolizumab) against a placebo on top of standard-of-care in more than 400 patients, all of whom had a history of previous surgery (approximately one in three had ≥3 surgeries) and required surgery due to severe symptoms and bigger polyps.

Aaron Royston, venBio

In­vest­ing in the time of coro­n­avirus: the good, the bad and the hope­ful, as biotech VC firms close funds worth $3B

Apart from disrupting biopharma R&D and regulatory timelines, the coronavirus pandemic has inevitably ravaged financial markets and eroded investor risk appetite. Investing in the time of coronavirus feels reckless, but if biotech venture funds are any indication, the time is ripe.

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Servi­er bags an an­ti­body spe­cial­ist in its lat­est on­col­o­gy M&A deal with plans to add the plat­form tech

Whatever Servier learned about Symphogen during their 2-year development alliance must have significantly whetted their appetite for an acquisition.

Paris-based Servier announced Friday that it has struck a deal to buy out the antibody expert. The acquisition comes 2 years after Servier acquired Shire’s cancer business for $2.4 billion. They’ve been working with Symphogen on a slate of programs, including some favs – PD-1, LAG3 and TIM3 — where they are looking to differentiate themselves from the more prominent drugs in these niches.

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GSK vac­cine chief heads for AIDS vac­cine ini­tia­tive; Pfiz­er en­lists Sue Desmond-Hell­mann to its board of di­rec­tors

→ Rip Ballou, who until very recently led vaccine research and development at GlaxoSmithKline, is joining the International AIDS Vaccine Initiative (IAVI) to lead its USAID-funded ADVANCE program. The program uses a network of researchers and institutions in Africa to help develop a vaccine for HIV. Ballou had worked at GSK since 2010 and has led global vaccine R&D since 2015. Prior to that he held posts at the Bill & Melinda Gates Foundation, a different post at GSK, Medimmune, and Walter Reed Army Institute of Research.  IAVI is led by Mark Feinberg, the former CSO of Merck Vaccines. 

Ahead of US IPO, Leg­end Biotech adds $150M, top-tier in­vestors to back CAR-T pipeline

Last month Nanjing Legend Biotech revealed that it sees, and was quietly planning for, a future as a public company in the US, separate but still tied to its former parent, Chinese CRO GenScript. It’s evidently a vision that enticed investors, drawing marquee names for a pre-IPO round.

The Series A fetched a whopping $150.5 million from Hudson Bay Capital Management, Lilly Asia Ventures, Vivo Capital, RA Capital Management and JJDC, the venture arm of J&J. The pharma giant has helped fund Legend’s CAR-T work with the $350 million upfront payment it handed over to partner on the lead BCMA program.

Drug dis­cov­ery in the age of coro­n­avirus

Developing new drugs is incredibly hard. That’s why, despite superhuman efforts from the industry, we’re still looking at 12-18 months minimum before we can realistically hope for a vaccine for Covid-19, and probably months before there’s a proven viable drug treatment.

But our increasing ability to begin to industrialize the drug discovery and development process through an engineering approach means that we have more hope for speeding up this process than ever before — and not just to defeat coronavirus, but to benefit the development of all new medicines in the future.