FDA slaps a hold on Sol­id Bio's gene ther­a­py for Duchenne MD in wake of safe­ty alert

Just a few weeks af­ter Sol­id Bio­sciences $SLDB put out an 11th hour no­tice that its gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy had been put on a par­tial clin­i­cal hold due to safe­ty con­cerns, just ahead of its $125 mil­lion IPO, the biotech is back Wednes­day evening with the news that the agency has now halt­ed the study in the wake of an ad­verse re­ac­tion to the ther­a­py by the first pa­tient in the tri­al.

The stock plunged 54% af­ter the news hit, sig­ni­fy­ing that this time around in­vestors aren’t quite so san­guine about safe­ty is­sues. But the pain didn’t end there. JP­Mor­gan, which helped on the IPO, turned bear­ish on the news and cau­tioned on the com­pa­ny’s prospects in the wake of a safe­ty is­sue that raised se­ri­ous doubts about Sol­id’s ex­ec­u­tive team. By mid-af­ter­noon on Thurs­day, the stock was down 63%, shear­ing off more than $600 mil­lion in val­ue.

“In our view, it will like­ly take com­pelling clin­i­cal da­ta to re­gain con­fi­dence in man­age­ment (not ex­pect­ed in the near-term),” said JP Mor­gan’s Anu­pam Ra­ma, ac­cord­ing to a Bloomberg re­port.  Ra­ma set the new price at $9, a sick­en­ing drop from the $16 price they launched at.

Sol­id Bio CEO Ilan Gan­ot

This is from Sol­id’s state­ment:

The first pa­tient dosed in the clin­i­cal tri­al was a non-am­bu­la­to­ry ado­les­cent who re­ceived 5E13 vg/kg of SGT-001 on Feb­ru­ary 14, 2018. Sev­er­al days af­ter ad­min­is­tra­tion the pa­tient was hos­pi­tal­ized due to lab­o­ra­to­ry find­ings that in­clud­ed a de­crease in platelet count fol­lowed by a re­duc­tion in red blood cell count and ev­i­dence of com­ple­ment ac­ti­va­tion. The pa­tient showed no signs or symp­toms of co­ag­u­lopa­thy (bleed­ing dis­or­der) and no rel­e­vant changes from base­line in liv­er func­tion tests. The pa­tient re­spond­ed well to med­ical treat­ment and is cur­rent­ly asymp­to­matic. All lab­o­ra­to­ry pa­ra­me­ters have ei­ther im­proved or re­turned to nor­mal, and he is con­tin­u­ing out­pa­tient as­sess­ments per pro­to­col.

The FDA is like­ly on high alert on Sol­id af­ter James Wil­son, a pi­o­neer in gene ther­a­py who de­vel­oped the AAV de­liv­ery tech that Sol­id is us­ing, re­signed from the ad­vi­so­ry board af­ter an an­i­mal study raised a red flag about po­ten­tial safe­ty is­sues when used at a high dose. Wil­son was alarmed af­ter a mon­key was killed by liv­er tox­i­c­i­ty af­ter be­ing ex­posed to a high dose AAV treat­ment, with ev­i­dence of mo­tor neu­ron dam­age in piglets. A num­ber of com­pa­nies use AAV tech in-li­censed for their work. But none of the oth­ers has been hit with a hold.

Sol­id CEO Ilan 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.

Some an­a­lysts sup­port­ing Sarep­ta $SRPT in the race to de­vel­op a gene ther­a­py for DMD are like­ly to glee­ful­ly sug­gest that Sol­id’s trou­bles are Sarep­ta’s boon. Sarep­ta has dosed a cou­ple of pa­tients now, look­ing for a one-time ther­a­py to help save boys from a dev­as­tat­ing ill­ness.

Gan­ot’s de­ci­sion to keep the par­tial hold out of the news while it was do­ing a road show on the IPO rais­es ques­tions about the lev­el of trans­paren­cy – or lack it – that al­lows pri­vate drug de­vel­op­ers to op­er­ate in se­cre­cy. Aside from no­ti­fy­ing re­searchers, reg­u­la­tors and pa­tients, a pri­vate de­vel­op­er is not re­quired to air safe­ty is­sues. The FDA cer­tain­ly won’t say any­thing. And we saw the ex­act same thing play out re­cent­ly when Unum filed their IPO, re­veal­ing two pa­tient deaths months ear­li­er and a clin­i­cal hold on the high dose of their lead cell ther­a­py, which they sub­se­quent­ly dumped.

The Cam­bridge, MA-based biotech says it’s work­ing with the FDA on get­ting the hold lift­ed.

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

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Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

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RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

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No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.

Is there a recipe for M&A suc­cess? The best and worst buy­out deals in the past decade of­fer some keys to suc­cess — and fail­ure

It’s not easy achieving a solid win in M&A in this industry. But if you follow a few simple guidelines, you may be able to increase your odds of success.
Geoffrey Porges and the team at SVB Leerink went about the “notoriously difficult” task of scoring the biopharma buyout of 2009 to 2019. Sizing up current and expected revenue from the products that were gained, they came up with the 5 winners:
Merck/Schering Plough
Bristol/Medarex
Gilead/Pharmasset
Sanofi/Genzyme
AstraZeneca/Acerta
It says a lot about the field that it’s much easier sorting out the 5 worst deals, though there’s also a lot more competition for that title, notes Porges. As picked by the analysts:
J&J/Actelion
Merck/Cubist
Alexion/Synageva
AbbVie/Stemcentrx
Gilead/Kite

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