FDA lifts clin­i­cal hold on Mus­tang Bio's lentivi­ral gene ther­a­py, paving the way for quick jump in­to piv­otal tri­al

Things were look­ing good for Mus­tang Bio’s MB-107 gene ther­a­py pro­gram. By ear­ly Sep­tem­ber, it had nabbed both rare pe­di­atric dis­ease and or­phan drug des­ig­na­tions, adding to the re­gen­er­a­tive med­i­cine ad­vanced ther­a­py des­ig­na­tion it al­ready scored at the FDA. It all bod­ed well for the piv­otal tri­al they were look­ing to start in new­ly di­ag­nosed in­fants with X-linked se­vere com­bined im­mun­od­e­fi­cien­cy.

There was just a prob­lem: The FDA had put a clin­i­cal hold on that tri­al in June.

Six months af­ter ex­ecs first alert­ed in­vestors to the hold, Mus­tang said they’ve fi­nal­ly been cleared to pro­ceed with dos­ing.

Reg­u­la­tors lift­ed the hold on the piv­otal Phase II tri­al on Jan. 28 af­ter re­view­ing a com­pre­hen­sive pack­age on chem­istry, man­u­fac­tur­ing and con­trols (CMC) — which was what raised con­cerns in the first place — sub­mit­ted in late De­cem­ber, Mus­tang added.

Manuel Litch­man

“The clin­i­cal out­comes ob­served in XS­CID pa­tients in the on­go­ing Phase 1/2 clin­i­cal tri­als con­tin­ue to be en­cour­ag­ing,” CEO Manuel Litch­man said in a state­ment. “It is es­pe­cial­ly grat­i­fy­ing to see the con­sis­tent safe­ty and ef­fi­ca­cy of our lentivi­ral vec­tor over the course of more than eight years since the first pa­tient was treat­ed at NI­AID in 2012.”

Mus­tang plans to start the tri­al in 2021, en­rolling 10 pa­tients whose da­ta would be com­piled with those from the 15 al­ready treat­ed in the cur­rent study. It’s pre­vi­ous­ly not­ed that in­ves­ti­ga­tors were con­tin­u­ing to ac­crue par­tic­pants while wait­ing for the hold to lift.

Af­ter treat­ment with MB-107, a one-time ther­a­py de­liv­er­ing the IL2RG gene in a lentivi­ral vec­tor, the 25 pa­tients will be com­pared with 25 “matched his­tor­i­cal con­trol pa­tients who have un­der­gone hematopoi­et­ic stem cell trans­plan­ta­tion” — the cur­rent treat­ment of choice that, while the­o­ret­i­cal­ly cu­ra­tive, can still leave pa­tients vul­ner­a­ble to in­fec­tions.

The pro­gram now be­ing de­vel­oped by Mus­tang came from St. Jude Chil­dren’s Re­search Hos­pi­tal, but the ap­proach has been test­ed for al­most two decades ei­ther as an al­ter­na­tive or fol­lowup to HSCT.

A sec­ond pro­gram, dubbed MB-207, is tar­get­ing that pop­u­la­tion who’s re­ceived trans­plants.

The piv­otal tri­al will be com­par­ing event-free sur­vival be­tween the two groups. Al­though it’s start­ing lat­er than orig­i­nal­ly ex­pect­ed, the com­pa­ny says it still an­tic­i­pates topline da­ta in the sec­ond half of 2022.

So­cial: Manuel Litch­man, PR Newswire

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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FDA re­view­ers en­dorse J&J sin­gle-dose shot, lay­ing path for 3rd US vac­cine

J&J’s single-dose vaccine may not have produced quite the stellar numbers of the mRNA shots, but it still won a ringing endorsement from FDA reviewers, who argued in briefing documents that it could provide robust protection against the still-raging virus.

The FDA confirmed that across just under 40,000 volunteers, the vaccine proved 66% effective at preventing symptomatic Covid-19, including 72% effective in the United States. Although that’s short of the 95% figures put up by Moderna and Pfizer-BioNTech, the shot was still 85% effective at stopping severe disease 28 days after administration. There were seven deaths in the placebo group — zero in the vaccine group.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Biden is look­ing be­yond Wood­cock or Sharf­stein for FDA com­mis­sion­er — re­port

Neither Janet Woodcock nor Joshua Sharfstein is likely to be nominated as the permanent FDA commissioner, Steve Usdin at BioCentury reports.

The White House is looking for alternatives to Woodcock, the acting chief and longtime CDER director, after opposition from several Democratic senators who are calling on others to block her nomination if her name is put forth, according to Usdin. Sharfstein, the former principal deputy FDA commissioner and current Johns Hopkins professor, is out of the running altogether.