Strug­gling Mar­i­nus gets a bump out of a pos­i­tive PhII snap­shot

Michael G. Chez, MD, Sut­ter Neu­ro­science In­sti­tute

A lit­tle more than six months since its stock price was crushed by a failed late-stage pro­gram for its lead­ing — and on­ly — drug, tiny biotech mi­cro­cap Mar­i­nus $MRNS saw its shares shoot up this morn­ing af­ter it of­fered a pos­i­tive Phase II snap­shot on a hand­ful of pa­tients who suf­fer from a rare ge­net­ic ail­ment.

In­ves­ti­ga­tors flipped the first card of its Phase II hand for ganax­olone in treat­ing CD­KL5, which trig­gers se­vere seizures and neu­ro­log­i­cal dam­age among the chil­dren af­flict­ed by it. One of four pa­tients had to drop out from a lack of re­sponse, but in­ves­ti­ga­tors say that the three oth­ers ex­pe­ri­enced a re­duc­tion in seizures rang­ing from 52% to 88%.

Pre­sent­ing da­ta at the an­nu­al meet­ing of the Amer­i­can Epilep­sy So­ci­ety, re­searchers al­so added seizure re­spons­es and EEG mea­sure­ments for three pa­tients — one with CD­KL5 and two with PCDH19 pe­di­atric epilep­sy — un­der­scor­ing a pos­i­tive re­sponse in cut­ting the rate of seizures from base­line.

In­vestors liked what they saw, dri­ving up shares about 40% in morn­ing trad­ing. It was a dif­fer­ent sto­ry last June, when the com­pa­ny’s shares dived on the news that ganax­olone had failed a late-stage study for adult fo­cal on­set seizures. Its mar­ket cap at the end of day on Fri­day was a frag­ile $22 mil­lion.

It’s ear­ly days yet for this study, which is de­signed to en­roll up to 10 pa­tients with each of CD­KL5 dis­or­der, Lennox Gas­taut Syn­drome and PCDH19 pe­di­atric epilep­sy.

The drug de­vel­op­ment pro­gram here is sim­i­lar to what Sage has been work­ing on, with a fo­cus on sta­tus epilep­ti­cus as well as post­par­tum de­pres­sion.

Michael G. Chez, the di­rec­tor of Pe­di­atric Neu­rol­o­gy Re­search and Pe­di­atric Epilep­sy, Sut­ter Neu­ro­science In­sti­tute, said:

“I am im­pressed with the re­spon­der rate and mag­ni­tude of seizure con­trol seen with ganax­olone in the ini­tial CD­KL5 pa­tients. The CGIs (clin­i­cal glob­al im­pres­sion scales) are con­sis­tent with seizure con­trol, with re­spon­ders show­ing ‘much im­proved’ un­der this scale. I look for­ward to fur­ther eval­u­at­ing these chil­dren and see­ing the fi­nal re­sults.”

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

AS­CO ab­stracts mint some ear­ly win­ners, but Io­vance, Spring­Works get hit by mas­sive loss­es

Before cancer-focused biotechs start their trek to the first in-person American Society of Clinical Oncology (ASCO) annual conference since the pandemic began, investors have taken a good look at the teasers for the data scheduled to be presented — and started placing bets.

With its power to confer overnight fame, ASCO is a stage where impressive or surprise debut performances can go a long way. On the other hand, disappointing details could be punishing.

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Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Levi Garraway, Roche CMO (Genentech)

Roche's CD20xCD3 does­n't beat Gen­mab at ORR, but sets bar for CR da­ta on lym­phoma drug

On its way to potentially becoming the first to market with a CD20xCD3 bispecific for aggressive lymphoma, Big Pharma’s largest R&D spender has some more data to pad its case with an FDA filing slated for later this year.

Roche dropped some more details from a Phase II expansion study of its fixed-duration glofitamab, to be presented at next week’s ASCO annual meeting, in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). The patients had received a median of three prior therapies.

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Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.