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.

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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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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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.

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.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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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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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.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.