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.”

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Atomwise CEO and co-founder Abraham Heifets (left) and co-founder Izhar Wallach

A cou­ple bil­lion for Ex­sci­en­tia was on­ly part of Sanofi's AI am­bi­tions, as the Big Phar­ma adds Atom­wise to the ta­ble

Sanofi made clear its AI ambitions were real at the beginning of this year when the Big Pharma took its drug discovery collaboration with Exscientia to the next level, inking a pact that could birth 15 drugs and deliver $5.3 billion to the UK partner.

Seven months later, the AI blueprint is far from over at the French Big Pharma, as another of the much-hyped drug discovery startups is coming to the table in a five-drug deal. Sanofi will pay Atomwise $20 million to kick off the hunt for up to five targets, which are aimed at leading to the creation of new small molecules. Another $1 billion is on the line — as are royalties — and the companies kept mum on the specific diseases or broader therapeutic areas of interest.

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Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.

Blaise Coleman, Endo International CEO

En­do files for Chap­ter 11 as it looks to fin­ish off its opi­oid lit­i­ga­tion

Irish drugmaker Endo International is entering into bankruptcy as it faces the weight of serious litigation related to its involvement in the opioid epidemic in the US.

The company has filed Chapter 11 proceedings in the US Bankruptcy Court for the Southern District of New York, with the company expected to file recognition proceedings in Canada, the UK and Australia. The company’s bankruptcy filing showed the company had assets and liabilities in the range of $1 billion to $10 billion.