Scott Braunstein, Marinus CEO

Af­ter strug­gling with its sole drug for years, Mar­i­nus rides pos­i­tive da­ta to a com­mer­cial­iza­tion deal in Eu­rope

Af­ter a se­ries of painful set­backs, Mar­i­nus had a re­birth of sorts last year when it of­fered a pos­i­tive late-stage pre­view of its sole drug, ganax­olone, in a ge­net­ic dis­or­der that caus­es ear­ly-on­set epilep­sy. Now, CEO Scott Braun­stein is rid­ing that suc­cess to an NDA sub­mis­sion and a col­lab­o­ra­tion with the Finnish phar­ma com­pa­ny Ori­on to com­mer­cial­ize the can­di­date in Eu­rope.

Ori­on is putting down around $30 mil­lion up­front (€25 mil­lion) and $115 mil­lion in biobucks (€97 mil­lion) for ex­clu­sive rights to mar­ket both the oral and IV for­mu­la­tions of ganax­olone in the EU, UK and Switzer­land for the treat­ment of CD­KL5 de­fi­cien­cy dis­or­der (CDD), tuber­ous scle­ro­sis com­plex (TSC) and re­frac­to­ry sta­tus epilep­ti­cus (RSE).

Braun­stein just sub­mit­ted an NDA for the oral ver­sion of the can­di­date, a GA­BA/A re­cep­tor mod­u­la­tor, in CDD — and he plans on fil­ing with Eu­ro­pean reg­u­la­tors in Q3 of this year. If all goes well, oral ganax­olone could hit the Eu­ro­pean mar­ket in mid-2022.

Braun­stein took the helm back in 2019, af­ter ganax­olone stum­bled in sev­er­al stud­ies for epilep­sy, Frag­ile X syn­drome and post­par­tum de­pres­sion.

“We were close to go­ing un­der a year ago,” he told End­points News last year. “We didn’t have a year’s worth of cash.”

Then came da­ta from the Phase III Marigold tri­al, which showed CDD pa­tients treat­ed with ganax­olone saw a 30.7% me­di­an re­duc­tion in 28-day ma­jor mo­tor seizure fre­quen­cy, com­pared to a 6.9% re­duc­tion for those on place­bo (p=0.0036). In an open-la­bel ex­ten­sion study, the ganax­olone pa­tients saw a 49.6% re­duc­tion. The drug was well-tol­er­at­ed, with the most fre­quent side ef­fect be­ing drowsi­ness, ac­cord­ing to the com­pa­ny.

Up­on read­ing out the topline da­ta, BAR­DA of­fered Mar­i­nus $21 mil­lion and a shot at $30 mil­lion more to sup­port the biotech’s work on an IV ver­sion for re­frac­to­ry sta­tus epilep­ti­cus. While the oral dos­ing is for chron­ic ill­ness, the IV for­mu­la­tion is de­signed to de­liv­er high dos­ing over a short in­ter­val for acute, se­vere med­ical con­di­tions, Braun­stein said.

“It’s pret­ty in­cred­i­ble to think we’ve been re­mote, we’ve raised over $200 mil­lion in cap­i­tal, we’ve now filed our first NDA, (and) we’re go­ing to an­nounce this Eu­ro­pean part­ner­ship,” he said. And they did it all while keep­ing up with re­mote yo­ga twice a week.

But it hasn’t been all smooth sail­ing. While the com­pa­ny was sched­uled to de­liv­er Phase III da­ta on RSE in the first half of 2022, that read­out has been pushed back to the sec­ond half of 2022. Braun­stein blamed the de­lay on the pan­dem­ic, which he says caused staff turnover and held things up at tri­al sites.

“I’m just re­al­ly proud of the team, how many achieve­ments they’ve con­tin­ued to hit in an in­cred­i­bly dif­fi­cult time,” Braun­stein said. “We’ve had a lit­tle bit of a stum­ble here in Q2, but (I’m) quite con­fi­dent we’re go­ing to bounce back.”

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Hedge fund jumps in with Avoro ac­tivists in an at­tempt to de­rail Mer­ck­'s $11B Ac­celeron buy­out

Avoro Capital, which made its bones blowing up the Seagen-Immunomedics deal and then selling the smaller biotech for $21 billion, is getting an assist in its quest to derail Merck’s $11 billion buyout of Acceleron $XLRN.

Wednesday morning one of Acceleron’s biggest investors joined the opposition. Darwin Global Management, a hedge fund which owns about 4% of Acceleron, blasted the Merck deal, saying the Big Pharma is getting the company for billions less than what it’s worth. Earlier, Holocene Advisers, reportedly a top-20 investor in Acceleron, said it would not tender its stock after criticizing the $180-per-share deal.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Suresh Katta, Saama CEO (via YouTube)

As AI con­tin­ues to en­tice Big Phar­ma, a Car­lyle-led drug­mak­er syn­di­cate shells out $430M for cloud com­put­ing play­er

The AI revolution permeating Big Pharma took a big financial step forward Wednesday, with VCs and major drugmakers coming together to acquire a cloud-focused company.

Led by the Carlyle Group, the investors will put up $430 million for a majority stake in Saama, a company that collects patient data to help speed along the drug development process. The investment arms of Pfizer, Merck, Amgen and McKesson all participated in the financing, in addition to other prominent life sciences VCs like Northpond.

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Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.