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.”

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

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Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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