Io­n­is re­ports a pos­i­tive PhI­II for volane­sors­en in rare meta­bol­ic dis­or­ders

Louis O’Dea, Akcea

In­ves­ti­ga­tors for Io­n­is Phar­ma­ceu­ti­cals $IONS say they gath­ered pos­i­tive ev­i­dence of ef­fi­ca­cy for their late-stage drug volane­sors­en in rare cas­es of meta­bol­ic dis­eases.

The drug pro­duced a mean re­duc­tion of 71.2% in triglyc­erides among the 113 pa­tients in the study who took the treat­ment over 13 weeks. That com­pares to a neg­li­gi­ble re­duc­tion among the place­bo arm of the study.

In ad­di­tion, 82% of pa­tients treat­ed with volane­sors­en achieved triglyc­eride lev­els less than 500 mg/dl af­ter 13 weeks of treat­ment, com­pared to 14% of place­bo-treat­ed pa­tients (p<0.0001).

There was one case of serum sick­ness record­ed in the study, which may have been drug re­lat­ed. Io­n­is, though, says it isn’t like­ly that the drug sparked that case.

This is one of sev­er­al late-stage stud­ies un­der­way for this drug, says Io­n­is, which plans to re­view its progress in ear­ly Jan­u­ary. The study re­cruit­ed pa­tients with rare meta­bol­ic dis­or­ders: fa­mil­ial chy­lomi­crone­mia syn­drome and fa­mil­ial par­tial lipody­s­tro­phy by tar­get­ing apolipopro­tein C-III with the an­ti­sense drug.

“The find­ings in COM­PASS re­in­force the ef­fi­ca­cy and safe­ty of volane­sors­en ob­served in Phase 2 stud­ies across mul­ti­ple pa­tient pop­u­la­tions, in­clud­ing FCS,” said Dr. Louis O’Dea, chief med­ical of­fi­cer for Akcea, a sub­sidiary of Io­n­is.  “No drug avail­able to­day has demon­strat­ed the mag­ni­tude of the triglyc­eride re­duc­tions ob­served with volane­sors­en.  These re­sults con­firm the po­ten­tial val­ue of tar­get­ing ApoC-III to low­er triglyc­erides in pa­tients who have high un­met need with po­ten­tial­ly life-threat­en­ing con­se­quences.”

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,800+ biopharma pros reading Endpoints daily — and it's free.

FDA ap­proves the third NMOSD drug in 18 months as Roche/Genen­tech beefs up its port­fo­lio of drugs for neu­ro­log­i­cal dis­or­ders

There were no FDA approved treatments for neuromyelitis optica spectrum disorder at the start of 2019. Now, as of Friday, there are three.

The latest entrant to the market is the Roche/Genentech drug satralizumab after US regulators gave it the thumbs up late Friday. An IL-6 inhibitor, the drug joins Alexion’s Soliris and AstraZeneca spinout Viela Bio’s Uplizna. The annual cost of satralizumab — which will hit the market as Enspryng — will be $190,000 for 13 doses, a Genentech spokesperson said, though the first year of treatment requires 15 doses and cost about $220,000.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,800+ biopharma pros reading Endpoints daily — and it's free.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,800+ biopharma pros reading Endpoints daily — and it's free.

US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,800+ biopharma pros reading Endpoints daily — and it's free.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,800+ biopharma pros reading Endpoints daily — and it's free.

Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 14. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,800+ biopharma pros reading Endpoints daily — and it's free.