Blue­bird’s gene ther­a­py for be­ta-tha­lassemia falls short of a cure, but still wows in 22 pa­tients

Af­ter go­ing back to the draw­ing board to come up with a bet­ter gene ther­a­py for be­ta-tha­lassemia, a dis­ease that in­ter­feres with the body’s abil­i­ty to pro­duce he­mo­glo­bin, in­ves­ti­ga­tors work­ing for blue­bird bio $BLUE have pro­duced some com­pelling da­ta to demon­strate that their ther­a­py can elim­i­nate or re­duce the need for blood trans­fu­sions.

But they don’t have a cure — yet.

Pub­lish­ing in the New Eng­land Jour­nal of Med­i­cine, the re­searchers re­port that they test­ed the treat­ment in 22 pa­tients. In the 9 pa­tients with the most se­vere form of the dis­ease, they re­port they were able to dra­mat­i­cal­ly re­duce the need for blood trans­fu­sions by 73% and elim­i­nate it for now in 3. Six of those pa­tients, though, have need­ed to con­tin­ue trans­fu­sions. And 12 of 13 pa­tients with a less se­vere form of the dis­ease cur­rent­ly no longer re­quire blood trans­fu­sions af­ter a sin­gle treat­ment — with a me­di­an of 26 months trans­fu­sion-free.

The in­ter­im da­ta great­ly ex­pands on the pa­tient ex­pe­ri­ences they can now bank on to demon­strate the ef­fi­ca­cy of the at­tempt at a once-and-done ther­a­py which af­fects hun­dreds of thou­sands of pa­tients. And the in­ves­ti­ga­tors have not aban­doned an at­tempt to find a com­plete cure for all.

“There is room for im­prove­ment, as we’d like to see the elim­i­na­tion of de­pen­den­cy on trans­fu­sion even for pa­tients with the most se­vere form of the dis­ease,” Har­vard Med­ical’s Philippe Leboulch told their in-house pub. “But there is al­so hope with pro­to­col mod­i­fi­ca­tions we have in­tro­duced in our Phase III tri­als.”

Blue­bird — run by CEO Nick Leschly — was forced to go back and change the man­u­fac­tur­ing process on Lenti­Glo­bin af­ter some un­even re­spons­es ear­ly on. Last sum­mer, the com­pa­ny came up with some en­cour­ag­ing da­ta on the first three pa­tients.

The gene ther­a­py is made by ex­tract­ing blood stem cells from pa­tients and in­sert­ing a func­tion­al be­ta-glo­bin gene in­to them, trans­plant­i­ng them af­ter a round of chemo to clear their bone mar­row.

Jerome Groop­man in The New York­er writes:

The chal­lenge now is find­ing ways to im­ple­ment a com­plex, po­ten­tial­ly life-sav­ing treat­ment in parts of the world where med­ical care is lim­it­ed. Es­tab­lished fa­cil­i­ties for au­tol­o­gous mar­row trans­plan­ta­tion al­ready ex­ist in many de­vel­oped na­tions, as do lab­o­ra­to­ries that can in­tro­duce a healthy glo­bin gene in­to stem cells. But, in some parts of the world where be­ta tha­lassemia is most com­mon, these fa­cil­i­ties do not yet ex­ist.

Con­trary to what the en­thu­si­asts may be pre­sent­ing to­day, there’s clear­ly room for im­prove­ment. And blue­bird is clear­ly not alone in the field try­ing to pro­duce a cure. CRISPR Ther­a­peu­tics, for ex­am­ple, has been us­ing its gene edit­ing tech to see if it can find a last­ing cure for the dis­ease. And those oth­er pro­grams, in­clud­ing ef­forts at Bel­licum, will like­ly con­tin­ue to see if they can leapfrog blue­bird with some­thing bet­ter.


blue­bird bio CEO Nick Leschly Get­ty

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 77,100+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 77,100+ biopharma pros reading Endpoints daily — and it's free.

FDA puts pe­di­atric aGVHD drug on pri­or­i­ty re­view lane — will they go vir­tu­al with the ad­comm?

Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.

With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.

Covid 19 roundup: Trump push­es his new fa­vorite, untest­ed drug; CRISPR out­lines crip­pling im­pact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 77,100+ biopharma pros reading Endpoints daily — and it's free.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 77,100+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 77,100+ biopharma pros reading Endpoints daily — and it's free.

ITeos nabs $125M as they prep Keytru­da com­bi­na­tion tri­al — if Covid-19 will let them

For iTeos, it turned out, $75 million could only last so long.

Two years after announcing their eye-catching Series B raise, the Belgian biotech is back with an even larger Series B-2: $125 million.

The now $200 million financing illustrates the vast capital available for those with promising new immuno-oncology compounds, particularly those that might be used in combination with existing therapies. In December, iTeos announced a collaboration with Merck to test its lead compound with Keytruda this year. The proceeds will push forward that trial and help fund the ongoing Phase I/II trials for that compound, EOS-850, and a second one, EOS-448.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 77,100+ biopharma pros reading Endpoints daily — and it's free.

Dai­ichi Sankyo sinks $200M in­to new gene ther­a­py tech from Ul­tragenyx

In a leap to the gene therapy space, Daiichi Sankyo has dropped $200 million to access Ultragenyx’s manufacturing technology, providing the rare disease biotech with plenty of cash and a stock boost amid a general cash crunch.

For $125 million in cash and a $75 million equity investment, Daiichi Sankyo has bought a non-exclusive license to the IP around two platforms with which it plans to develop AAV-based gene therapy products. The Japanese pharma is purchasing the stock $RARE at $60 per share, more than a third above its current price of $44.43.

It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).