Gala­pa­gos spot­lights an 'ex­cit­ing' round of PoC da­ta for IPF drug, shares surge

Gala­pa­gos $GLPG says it got the proof-of-con­cept da­ta re­searchers were look­ing for from their mid-stage tri­al of GLPG1690 for id­io­path­ic pul­monary fi­bro­sis and will now push for­ward as planned in­to a piv­otal pro­gram.

Fast on the heels of a ri­otous re­cep­tion for Fi­bro­Gen’s PoC round for a ri­val IPF drug, the Bel­gian biotech says their drug clear­ly sta­bi­lized a small group of pa­tients with the dis­ease while pa­tients in the place­bo arm con­tin­ued to de­cline.

Piet Wiger­inck, Gala­pa­gos. Cred­it: F. Kalk­man

The forced vi­tal ca­pac­i­ty (FVC) of lungs on the drug edged up 8 mL in 23 IPF pa­tients while the place­bo group saw a mean de­cline from base­line of 87 mL.

The biotech al­so spot­light­ed “a clear re­duc­tion of serum LPA18:2, a bio­mark­er for au­to­tax­in in­hi­bi­tion.”

In­vestors went nuts over the Fi­bro­Gen re­sults ear­li­er. Gala­pa­gos didn’t see the same eye-open­ing re­sponse, but its stock soared as well, spik­ing more than 20% af­ter bounc­ing around con­sid­er­ably af­ter the mar­ket closed.

Im­pe­r­i­al Pro­fes­sor To­by Ma­her cheered the re­sults:

Gala­pa­gos’ re­sults with GLPG1690 are ex­treme­ly ex­cit­ing and ex­ceed those of pre­vi­ous stud­ies. This brings hope to pa­tients with id­io­path­ic pul­monary fi­bro­sis that new ef­fec­tive treat­ment may be on the hori­zon. Im­por­tant­ly, some pa­tients even showed an in­crease of lung func­tion with­in on­ly 12 weeks of treat­ment, and the drug was well tol­er­at­ed. The re­sults from FLO­RA beg the ques­tion how pa­tients will fare with longer treat­ment. I urge Gala­pa­gos and the IPF com­mu­ni­ty to progress to the next phase of clin­i­cal tri­als as rapid­ly as pos­si­ble.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Mene Pangalos (AstraZeneca via YouTube)

As­traZeneca shuts the PhI­II door for Ion­is' PC­SK9 drug de­spite pos­i­tive PhI­Ib

When Ionis and AstraZeneca unveiled the first round of mid-stage data for their antisense PCSK9 drug, Mene Pangalos, AstraZeneca’s EVP of biopharmaceuticals R&D, underscored the drug’s “potential best-in-class efficacy profile.”

But now that the second batch is in, it appears AZD8233 isn’t hitting the mark after all.

Ionis announced Friday morning that although the candidate, also dubbed ION449, met the primary endpoint in the Phase IIb SOLANO trial, its partners at AstraZeneca have decided not to move it into Phase III studies because the “results did not achieve pre-specified efficacy criteria.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: Bio­gen throws it­self back in­to mud­dled da­ta ar­gu­ments with more de­tails on its an­ti­sense ALS drug

With a highly watched FDA decision deadline coming in late January, Biogen and Ionis dropped the full data on the Phase III study of their ALS drug tofersen in the New England Journal of Medicine on Wednesday.

Biogen is looking for approval for tofersen in a very small subset of ALS patients — some 2%, according to the paper — who have a SOD1 gene mutation, which has previously been linked to ALS. Tofersen is meant to reduce levels of mutant SOD1 proteins.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.