Boehringer In­gel­heim touts pre­ven­tion re­sults in rarest form of pso­ri­a­sis

Boehringer In­gel­heim un­corked some pos­i­tive re­sults sug­gest­ing that Spe­vi­go can help pre­vent flare-ups in pa­tients with a se­vere form of pso­ri­a­sis, months af­ter the drug was ap­proved to treat ex­ist­ing flares.

Spe­vi­go, an IL-36R an­ti­body al­so known as spesolimab, met its pri­ma­ry and a key sec­ondary end­point in the Phase IIb EFF­ISAY­IL 2 tri­al in pa­tients with gen­er­al­ized pus­tu­lar pso­ri­a­sis (GPP), Boehringer an­nounced on Mon­day. While the com­pa­ny is keep­ing the hard num­bers un­der wraps un­til lat­er this year, it said in a news re­lease that it an­tic­i­pates shar­ing the re­sults with reg­u­la­tors.

“Painful GPP flares can oc­cur sud­den­ly, es­ca­late quick­ly, and may re­quire ur­gent hos­pi­tal care leav­ing peo­ple anx­ious and un­cer­tain about what the fu­ture might hold,” Carinne Brouil­lon, a mem­ber of the com­pa­ny’s board of man­ag­ing di­rec­tors re­spon­si­ble for the Hu­man Phar­ma Busi­ness Unit, said in the news re­lease.  The com­pa­ny was not im­me­di­ate­ly avail­able for com­ment.

GPP is the rarest form of pso­ri­a­sis, and is char­ac­ter­ized by red and in­flamed patch­es of skin, as well as small pus­tules. The con­di­tion is some­times mis­tak­en for an in­fec­tion, and un­til Spe­vi­go’s ap­proval back in Sep­tem­ber, there were no drugs of­fi­cial­ly ap­proved to treat it. In se­ri­ous cas­es, the con­di­tion can lead to heart fail­ure, re­nal fail­ure and sep­sis.

While most pa­tients en­rolled in a Phase II piv­otal tri­al had a “high or very high” den­si­ty of pus­tules, 54% showed no vis­i­ble pus­tules af­ter a week of treat­ment with Spe­vi­go, com­pared to 6% of those on a place­bo, ac­cord­ing to Boehringer.

The EFF­ISAY­IL 2 tri­al showed a “sig­nif­i­cant pre­ven­tion of gen­er­al­ized pus­tu­lar pso­ri­a­sis (GPP) flares for up to 48 weeks” in ado­les­cents and adults, the com­pa­ny said on Mon­day.

Anap­tys­Bio is de­vel­op­ing its own IL-36R an­ti­body, im­si­dolimab, for the same in­di­ca­tion, though it said last year that it would look to out-li­cense the can­di­date be­fore a po­ten­tial FDA ap­proval. Ac­cord­ing to the com­pa­ny’s web­site, topline da­ta are ex­pect­ed in the fourth quar­ter of this year.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

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.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Ribbon cutting ceremony for Thermo Fisher's new cell therapy manufacturing site in San Francisco

Ther­mo Fish­er moves on cam­pus with new cell man­u­fac­tur­ing site in San Fran­cis­co

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.

Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

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.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Man­u­fac­tur­ing roundup: Catal­ent to pro­duce low-cost ver­sion of nalox­one; CSL opens R&D site

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.

Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

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.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.