Ipsen splurges $25M on its sec­ond rare dis­ease drug pact of the year, with more on the way

Eight months af­ter reel­ing in Clemen­tia and their drug palo­varotene in a $1.3 bil­lion buy­out deal, David Meek has an­oth­er rare dis­ease ther­a­py to add to the pipeline at Ipsen.

David Meek

The Paris-based biotech (EPA: $IPN) is fronting $25 mil­lion in cash to ac­quire world­wide rights to Blue­print’s BLU-782, an ALK2 in­hibitor de­signed to fight an ul­tra-rare ge­net­ic con­di­tion called fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va, or sim­ply FOP. And there’s the full slate of $510 mil­lion more for mile­stones run­ning through de­vel­op­ment, reg­u­la­to­ry as well as com­mer­cial.

That didn’t help Blue­print’s share price, though. The stock was in the red ahead of the bell with in­vestors like­ly a lit­tle edgy over the lim­it­ed amount of cash in­volved and a low­er like­li­hood of a buy­out that would have jacked up their eq­ui­ty val­ue con­sid­er­ably.

At the be­gin­ning of this year, BLU-782 had been teed up as the next drug to beef up the biotech’s pipeline, with plans to run a Phase I in healthy vol­un­teers and then piv­ot to Phase II in the first half of next year.

Loxo On­col­o­gy $LOXO — ac­quired by Eli Lil­ly — and Blue­print Med­i­cines $BPMC have been duk­ing it out with their stud­ies on ri­val RET in­hibitors, with the Loxo drug typ­i­cal­ly com­ing out ahead in the com­par­isons. Back in June Blue­print al­so sub­mit­ted an NDA for avapri­tinib, a treat­ment for adults with PDGFRA Ex­on 18 mu­tant gas­troin­testi­nal stro­mal tu­mors (GIST), re­gard­less of pri­or ther­a­py, and fourth-line GIST.

Ipsen, mean­while, seems hap­py to pur­sue more rare dis­ease drugs as Meek po­si­tions the com­pa­ny along some fa­mil­iar lines in the in­dus­try. Rare dis­eases — with their small tri­als and ac­cel­er­at­ed re­views — has be­come an at­trac­tive space for a broad swath of biotechs around the world.

So­cial im­age: David Meek, Ipsen Group via Youtube

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

Endpoints Premium

Premium subscription required

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

Michael Gladstone, partner at Atlas Venture

At­las rais­es new $400M fund amid spree of VC rais­es. Here’s what they’ll spend it on

You can add another few hundred million to the now Montana-sized reservoir of cash biotech VCs have raised since the WHO declared Covid-19 a pandemic.

Atlas Venture, the prominent Kendall Square incubator, has raised $400 million for its twelfth biotech fund, their first in 3 years. After a string of mammoth new raises from other major VCs in April and May, the total pot now stands between $5 billion and $6 billion, depending on how you slice it.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.