Duchenne MD dad piv­ots from last-minute alert on FDA's par­tial hold to $125M IPO for Sol­id Bio

In most IPOs, risk fac­tors can range from any­thing from “our lead drug may not work” to the threat of famine and plague — they’re in­tend­ed to cov­er vir­tu­al­ly every con­tin­gency.

Ilan Gan­ot

But as Ilan Gan­ot was out drum­ming up sup­port for his $125 mil­lion IPO for Sol­id Bio­sciences in re­cent months, there was one key risk fac­tor that hadn’t been in­clud­ed. The FDA had put its lead drug on par­tial clin­i­cal hold in No­vem­ber, with reg­u­la­tors re­fus­ing to al­low re­searchers to start test­ing the high dose.

On­ly the low dose was green-light­ed for the tri­al, the com­pa­ny dis­closed in a tardy amend­ment to the IPO to­day, but the high dose was stymied un­til Sol­id could “sub­mit ad­di­tion­al CMC in­for­ma­tion that demon­strates that man­u­fac­tur­ing ca­pac­i­ty and prod­uct at­trib­ut­es can sup­port the high-dose group.”

And that’s not all.

Ear­li­er this month the biotech not­ed that gene ther­a­py pi­o­neer James Wil­son from Penn had re­signed from their sci­en­tif­ic ad­vi­so­ry board due to ris­ing safe­ty con­cerns re­lat­ed to high dos­ing us­ing the vec­tor he had de­vel­oped.

Re­cent­ly, James M. Wil­son, M.D., Ph.D., re­signed from our Sci­en­tif­ic Ad­vi­so­ry Board cit­ing emerg­ing con­cerns about the pos­si­ble risks of high sys­temic dos­ing of AAV. If in the fu­ture we are un­able to demon­strate that any such ad­verse events were not caused by the ad­min­is­tra­tion process or re­lat­ed pro­ce­dures, the FDA, the Eu­ro­pean Com­mis­sion, the EMA or oth­er reg­u­la­to­ry au­thor­i­ties could or­der us to cease fur­ther de­vel­op­ment of, or de­ny ap­proval of, SGT-001 or our oth­er prod­uct can­di­date for any or all tar­get­ed in­di­ca­tions.

The news, though, didn’t hit un­til Sol­id Bio was wrap­ping up the pric­ing. Sol­id has been plan­ning to sell about 6 mil­lion shares at $16 to $18 a share, with a mar­ket val­u­a­tion of rough­ly $550 mil­lion. Thurs­day evening, Sol­id put out an an­nounce­ment that they had sold 7.8 mil­lion shares at $16 apiece.

Their suc­cess comes amid a wave of fresh hits on the IPO front. Men­lo Ther­a­peu­tics bagged $119 mil­lion in an up­sized of­fer­ing to get the sea­son un­der­way on Thurs­day. And right on the heels of the move by Sol­id Bio, resTOR­bio raised $85 mil­lion and Ar­mo gar­nered $86 mil­lion, bring­ing the to­tal haul from 4 IPOs in two days to $415 mil­lion, which is sure to in­spire plen­ty more new IPOs in Q1.

Gan­ot — a for­mer JP Mor­gan in­vest­ment banker — has made much of the fact that he’s a Duchenne MD dad out to find a gene ther­a­py that could cure the lethal, rare dis­ease. By in­tro­duc­ing a syn­thet­ic dy­s­trophin trans­gene con­struct, called mi­crody­s­trophin, via a vi­ral vec­tor, the com­pa­ny hopes to prove it can do what Sarep­ta and oth­ers have been grop­ing for with one de­ci­sive in­ter­ven­tion. And he had at­tract­ed some heavy­weight back­ers, in­clud­ing RA Cap­i­tal and their col­leagues at Bain.

Now their new risk fac­tor in­cludes the note that the drug may fail if they can’t get the FDA to lift the hold, which they were in­formed of at least two months ago.

I not­ed at the be­gin­ning of Jan­u­ary that the IPO Gan­ot filed al­so failed to lay out ex­act­ly who owned what in the com­pa­ny, an odd omis­sion for some­one shoot­ing to raise that much cash.

The lat­est up­dates in­clude the fact that Gan­ot owns 4.3% of the com­pa­ny with JPMC Strate­gic In­vest­ments in for 9% and Per­cep­tive at 8.7%.

Gan­ot him­self will earn a base salary of $450,000 this year, with a shot at a $200,000 bonus. And his wife is al­so work­ing for the com­pa­ny, with a salary that’s un­der $200,000.

The stock will trade as $SLDB, with every­one look­ing to see where it heads on Fri­day.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.