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.”

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

With lumasir­an on the FDA's doorstep, Al­ny­lam reads out new PhI­II da­ta in PH1

Just over a month away from its December PDUFA date, Alnylam flaunted new data from two Phase III studies to back lumasiran in primary hyperoxaluria type 1 (PH1), a rare liver condition.

The Cambridge, MA-based biotech snagged a priority review for the candidate back in June, and got positive feedback from the EMA’s Committee for Medicinal Products for Human Use just last week. Lumasiran uses RNA interference (RNAi) to silence the gene for glycolate oxidase, an enzyme used in the production of oxalate.

Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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News brief­ing: UK biotech 4D phar­ma heads for Nas­daq via SPAC; Dr. Red­dy's shuts down man­u­fac­tur­ing af­ter cy­ber­at­tack

Another pharma company is intending to use a SPAC to join the Nasdaq.

4D pharma, a UK-based biotech, is reverse-merging with a blank check company in a deal worth up to $37.6 million. The move will give 4D pharma a new Nasdaq ticker, which will be $LBPS, using the American Depositary Share program.

As a result of the move, 4D pharma will gain $14.6 million in cash held by the blank check company, dubbed Longevity. The merger is expected to be completed in early 2021, after which shares will be immediately tradeable.

Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Jason Kelly, Gingko Bioworks CEO (Mike Blake/Reuters via Adobe)

Ex­clu­sive: Eye­ing big Covid-19 test­ing ex­pan­sion, Gink­go rolls out 50M rapid anti­gen di­ag­nos­tics

In what they hope will be a key part of an extensive effort to boost Covid-19 testing in the US, Ginkgo Bioworks is acquiring and distributing 50 million rapid antigen tests that can potentially be used for virus surveillance in schools and communities and for quick, on the ground diagnoses.

The tests, developed by SD Biosensor, are in line with proposals from the Rockefeller Foundation and Harvard epidemiologists, among others, to blanket the country with fast, low-cost tests. Although not yet authorized in the US, they are a key part of testing efforts from the Bill and Melinda Gates Foundation, who announced plans last month to distribute 120 million of them in low and middle income countries. Roche has commercialized the diagnostic in Europe.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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