Jeremy Levin via YouTube

Ovid cuts its loss­es on con­tro­ver­sial An­gel­man syn­drome drug, mak­ing its crush­ing de­vel­op­ment halt per­ma­nent

Af­ter a tur­bu­lent cou­ple of years, Ovid is of­fi­cial­ly do­ing away with a pro­gram it had once cham­pi­oned in the face of heavy an­a­lyst skep­ti­cism.

Ovid has dis­con­tin­ued de­vel­op­ment of OV101, or gabox­adol, in An­gel­man syn­drome and will not pur­sue fur­ther clin­i­cal tri­als for Frag­ile X syn­drome, the com­pa­ny an­nounced Mon­day morn­ing. The news comes af­ter Ovid had pre­vi­ous­ly paused de­vel­op­ment in An­gel­man when the com­pound flunked a Phase III tri­al in De­cem­ber.

Ovid $OVID shares, which were crushed af­ter that De­cem­ber read­out, were more mut­ed Mon­day — the stock was down about 2% in ear­ly morn­ing trad­ing. Ovid did not re­spond to a re­quest for com­ment Mon­day morn­ing.

In a re­lease, the biotech said the de­ci­sion to shut down OV101 was based on the “to­tal­i­ty” of da­ta from at least four clin­i­cal tri­al pro­grams. Though the com­pound had demon­strat­ed safe­ty and tol­er­a­bil­i­ty, the ef­fi­ca­cy proved lack­ing and ul­ti­mate­ly prompt­ed the dis­con­tin­u­a­tion.

OV101 it­self is a delta-se­lec­tive GABAA re­cep­tor ag­o­nist and was one of the first can­di­dates put in­to test­ing for the treat­ment of An­gel­man syn­drome, a ge­net­ic dis­or­der where a mu­ta­tion in an en­zyme in the brain can lead to a wide range of dif­fer­ent symp­toms. The gene in­volved is UBE3A: The mu­ta­tion oc­curs in the uterus and af­fects every cell in the brain as it de­vel­ops.

The big tu­mult over the ex­per­i­men­tal drug be­gan back in 2018, when Ovid re­port­ed Phase II re­sults. Though the twice-dai­ly drug arm whiffed bad­ly on sta­tis­ti­cal sig­nif­i­cance, CEO Je­re­my Levin pushed for­ward with a Phase III, ar­gu­ing that the true ef­fi­ca­cy mea­sure came in a less­er-known sec­ondary mea­sure of physi­cian-rat­ed clin­i­cal glob­al im­pres­sions of im­prove­ment (CGI-I).

Ovid and Levin con­tin­ued to in­sist that they nailed this end­point, but an­a­lysts and in­vestors saw it as a fluke that couldn’t be repli­cat­ed. The skep­tics even­tu­al­ly proved cor­rect as OV101 again missed in its Phase III study fo­cus­ing on CGI-I as the place­bo group out­per­formed the drug arm. OV101 al­so whiffed on all sec­ondary end­points in the Phase III study.

At the time, Ovid said it would pause de­vel­op­ment of the pro­gram as it sought dis­cus­sions with the FDA to “de­ter­mine next steps, if any.” Not on­ly did it mark a set­back for Ovid but al­so for pa­tients with An­gel­man syn­drome, for which there re­main no FDA-ap­proved ther­a­pies.

With OV101 out of the pic­ture, Ovid is piv­ot­ing to­ward its ear­li­er-stage as­sets, first and fore­most be­ing OV882. A short hair­pin RNA ther­a­py tar­get­ing UBE3A gene ex­pres­sion in neu­rons, this pro­gram will con­tin­ue Ovid’s ef­forts in An­gel­man syn­drome, the com­pa­ny said Mon­day. It’s still in very ear­ly stages, how­ev­er, hav­ing not yet hit the pre­clin­i­cal phase.

There’s al­so the OV935, or soti­cle­stat, pro­gram for de­vel­op­men­tal and epilep­tic en­cephalopathies, which Ovid li­censed to Take­da at the end of March. Ovid pulled in a $196 mil­lion up­front pay­ment for that pro­gram, and is el­i­gi­ble for up to $660 mil­lion in mile­stones and up to 20% roy­al­ties on po­ten­tial sales. The com­pa­ny has no fur­ther fi­nan­cial oblig­a­tions for this pro­gram, it not­ed Mon­day.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

UP­DAT­ED: Bio­gen push­es in a fresh stack of chips and starts prep­ping a glob­al R&D game plan af­ter watch­ing the cards turn on ear­ly throm­bolyt­ic da­ta

After patiently steering through a decade-long journey for its early-stage clinical work, a small Tokyo biotech has clinched a deal to out-license its lead thrombolytic agent to US heavyweight Biogen — which sees a potentially game-changing impact on the clot-busting field after taking a careful look at some upbeat Phase IIa data.

Three years after Biogen anted up $4 million to gain an option on the drug from TMS, the big US biotech is making a small bet to beef up its stroke portfolio. The BD team inked a deal to go ahead and grab rights to the drug for $18 million, with another $335 million in milestone cash on the table for a successful outcome.

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Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Alvotech takes Ab­b­Vie to court over al­leged patent 'mine­field' sur­round­ing megablock­buster Hu­mi­ra

AbbVie has so far been successful in shooing away competition to its megablockbuster Humira, deploying a number of patents and settlements to keep biosimilars off the US market until 2023. But one Icelandic drugmaker doesn’t want to wait — and on Tuesday, it filed a lawsuit challenging what it called a patent “minefield.”

Alvotech has accused AbbVie of trying to “overwhelm” and “intimidate” it with “an outrageous number of patents of dubious validity,” according to court documents. The company is currently seeking approval for its Humira copycat AVT02, which AbbVie says would infringe upon 62 patents.