RNA play­er Stoke Ther­a­peu­tics said Wednes­day that the FDA par­tial­ly lift­ed a clin­i­cal hold on a Phase I/IIa study in chil­dren and ado­les­cents with Dravet syn­drome, al­low­ing the com­pa­ny to in­crease the test­ed dose of its lead drug.

Dawn Kalmar

Stoke spokesper­son Dawn Kalmar tells End­points News the hold dates back to March 2020 when the com­pa­ny first got clear­ance to start Phase I/II stud­ies test­ing can­di­date STK-001 in Dravet syn­drome pa­tients. The de­ci­sion to put the par­tial hold in place was based on pre­clin­i­cal da­ta, Kalmar added.

“At the time back in March of ‘20, they had lim­it­ed dos­ing to 20 mil­ligrams, mul­ti­ple dos­es of 20 mil­ligrams. Then in Oc­to­ber of 2020, they al­lowed us to move to a 30 mil­ligram dose. Then in Sep­tem­ber of 2021, they al­lowed us to move to a 45 mil­ligram dose — and now we’re an­nounc­ing that they’re al­low­ing us to move to a 70 mil­ligram dose, a sin­gle 70 mil­ligram dose,” Kalmar said.

STK-001 is an an­ti­sense oligonu­cleotide can­di­date in the same class as Io­n­is and Bio­gen’s nusin­ersen, al­so known as Spin­raza, for spinal mus­cu­lar at­ro­phy.

Kalmar added that the study has been on­go­ing since the hold, dos­ing pa­tients in line with what the FDA’s hold re­quired. And af­ter turn­ing in da­ta from those dose lev­els, the FDA al­lowed Stoke to go to the next dos­ing lev­el.

Dravet syn­drome is a rare ge­net­ic epilep­sy dis­or­der char­ac­ter­ized by fre­quent seizures in kids un­der 1 year of age. On top of de­vel­op­men­tal de­lays and sleep­ing dis­or­ders as ad­di­tion­al symp­toms, 10-20% of those with Dravet syn­drome do not live be­yond age 10.

The biotech has been con­duct­ing a sis­ter study in the UK called AD­MI­RAL — which was ini­tial­ly ap­proved at high­er dos­es.

“We’ve been treat­ing pa­tients there with 30, and 45, and 70 mil­ligrams in the UK,” Kalmar added.

De­spite the up­date, shares of $STOK fell more than 8% af­ter the mar­ket opened Wednes­day morn­ing.

Though some of the hold has been lift­ed, an­oth­er part re­mains on the ex­ten­sion study, which is capped at 30 mg re­peat­ed dos­ing. The biotech is hop­ing it can fin­ish the tri­al and move in­to Phase III some­time next year.

Stoke read out da­ta from the tri­al last No­vem­ber, show­ing that a sub­group of six pa­tients ex­am­ined at 45 mg — who had all re­ceived three dos­es — had a me­di­an re­duc­tion of 55% in con­vul­sive seizure fre­quen­cy.

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Catalent will be manufacturing a low-cost version of the opioid overdose treatment naloxone as part of a contract with Harm Reduction Therapeutics.

Catalent plans to manufacture the treatment at its facility in Morrisville, NC. No financial details on the deal were disclosed.

Harm Reduction was granted priority review status for the NDA on its spray last year. The company has been working on a naloxone product since 2017. It is anticipating approval in July of this year and a US launch in early 2024.

Last year, South African-based vaccine manufacturer Aspen Pharmacare was facing reports that it had not received a single order for its manufactured Covid-19 shots and that manufacturing lines were sitting idle. But now the vaccine producer is looking to turn things around.

Aspen’s disclosure of its financial results in March unveiled that manufacturing revenue had decreased by 12% to R 603 million ($33.8 million), which Lorraine Hill, Aspen Group’s COO, said is attributable to lower Covid vaccine sales.

Thermo Fisher Scientific is putting down more roots in the Bay Area.

The manufacturer opened the doors to a new cell therapy manufacturing facility next to the University of California-San Francisco Medical Center’s Mission Bay campus and on the university’s campus.

UCSF and Thermo Fisher have had a partnership since 2021, with the new site focusing on manufacturing cell therapeutics for certain cancers, including glioblastoma and multiple myeloma. The new site plans to use Thermo Fisher’s expertise in manufacturing services to help UCSF accelerate the development of cell therapies and eventually get them into the clinic, said Dan Herring, the general manager of cell therapy services at Thermo Fisher, in an interview with Endpoints News.