No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

No­var­tis’ sick­le cell drug, ap­proved in 2019 and brand­ed as Adakveo, has failed an on­go­ing Phase III, ac­cord­ing to pre­lim­i­nary re­sults.

The Swiss phar­ma gi­ant un­veiled ear­ly da­ta from the on­go­ing STAND Phase III study on Fri­day, say­ing that crizan­l­izum­ab showed no sta­tis­ti­cal­ly sig­nif­i­cant dif­fer­ence be­tween the drug at two dif­fer­ent dose lev­els com­pared to place­bo in an­nu­al­ized rates of va­so-oc­clu­sive crises that lead to a health­care vis­it over the first year since be­ing ran­dom­ized in­to the tri­al.

Va­so-oc­clu­sive crises are high­ly painful events that oc­cur when sick­led red blood cells block blood flow to the point that tis­sues be­come de­prived of oxy­gen. It can al­so cause or­gan dam­age and in­crease chances of dy­ing.

No­var­tis said in a state­ment that the find­ings are in­con­gru­ous with pre­vi­ous tri­al re­sults, point­ing to the SUS­TAIN tri­al that showed crizan­l­izum­ab at 5.0mg/kg as su­pe­ri­or to place­bo.

Crizan­l­izum­ab was ap­proved by the FDA in No­vem­ber 2019 to re­duce the fre­quen­cy of va­so-oc­clu­sive crises. The drug was picked up from its Se­lexys buy­out for up to $665 mil­lion in 2016. Al­most a year af­ter the FDA ap­proval, the EMA grant­ed con­di­tion­al mar­ket­ing au­tho­riza­tion to pre­vent re­cur­rences of these events in pa­tients aged 16 years and old­er with sick­le cell dis­ease.

A No­var­tis spokesper­son told End­points News in an email:

Our pri­or­i­ty now is to thor­ough­ly re­view the full da­ta set of the STAND study and we are work­ing with reg­u­la­tors glob­al­ly, in­clud­ing the EMA and FDA, and tri­al in­ves­ti­ga­tors to de­ter­mine the ap­pro­pri­ate next steps. Any de­ci­sions on the over­all crizan­l­izum­ab de­vel­op­ment pro­gram will be made in co­or­di­na­tion with tri­al in­ves­ti­ga­tors and reg­u­la­to­ry au­thor­i­ties. Cur­rent­ly, all oth­er on­go­ing stud­ies are con­tin­u­ing as per pro­to­col.

Sick­le cell dis­ease has been at­tract­ing more and more at­ten­tion in re­cent years, but it’s had its pit­falls. Gly­coMimet­ics, a for­mer Pfiz­er part­ner, had a can­di­date that failed to meet both the pri­ma­ry and sec­ondary end­points in a Phase III tri­al in 2019, re­sult­ing in the al­liance get­ting cut.

More re­cent­ly in De­cem­ber, Ed­i­tas Med­i­cine re­port­ed some ear­ly-stage da­ta in two pa­tients suf­fer­ing from sick­le cell dis­ease, not­ing that nei­ther pa­tient suf­fered va­so-oc­clu­sive events in ini­tial months of fol­lowup af­ter be­ing treat­ed with a cell ther­a­py can­di­date.

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

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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.

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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.

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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.