FDA slams door to piv­otal tri­al for bub­ble boy dis­ease gene ther­a­py as Mus­tang Bio runs in­to an­oth­er hold

Mus­tang Bio is in fa­mil­iar ter­ri­to­ry, but that isn’t a place it nec­es­sar­i­ly wants to be.

The FDA has placed a hold on Mus­tang Bio’s piv­i­tol tri­al for its gene ther­a­py to treat pa­tients with bub­ble boy dis­ease, cit­ing is­sues sur­round­ing chem­istry, man­u­fac­tur­ing and con­trols clear­ance. It’s the sec­ond hold due to CMC is­sues the com­pa­ny has re­ceived in rough­ly 18 months.

An in­ves­ti­ga­tion­al new drug ap­pli­ca­tion was sub­mit­ted in De­cem­ber 2021. If grant­ed an IND, a Phase II study will then as­sess safe­ty, tol­er­a­bil­i­ty and ef­fi­ca­cy of MB-207. If ap­proved by the FDA, the ther­a­py would one day be el­i­gi­ble for a rare pe­di­atric dis­ease vouch­er.

The ther­a­py, known as MB-207, has been pre­vi­ous­ly grant­ed or­phan drug and rare pe­di­atric dis­ease des­ig­na­tions. The lentivi­ral ther­a­py is for those pa­tients who have al­ready been treat­ed with a hematopoi­et­ic stem cell trans­plan­ta­tion, and need more treat­ment.

Bub­ble boy dis­ease is of­fi­cial­ly known as X-linked se­vere com­bined im­mun­od­e­fi­cien­cy. It’s a rare ge­net­ic dis­or­der that oc­curs in 1 per 225,000 births, and is char­ac­ter­ized by the lack of func­tion of key im­mune cells. That leaves chil­dren with a se­vere­ly com­pro­mised im­mune sys­tem, and can lead to the ear­ly death of chil­dren. Many who are af­fect­ed by the dis­ease are un­able to es­tab­lish T cell im­mu­ni­ty.

Mus­tang was pre­vi­ous­ly is­sued a hold for MB-107, which is cur­rent­ly in Phase I/II tri­als at St Jude, UCSF Be­nioff Chil­dren’s Hos­pi­tal in San Fran­cis­co and Seat­tle Chil­dren’s Hos­pi­tal in new­ly di­ag­nosed in­fants un­der the age of two. In June 2021, the progress of MB-107 was de­layed, but about a year ago, the hold was cleared up, and reg­u­la­tors al­lowed the Phase II tri­al to pro­ceed af­ter re­ceiv­ing a com­pre­hen­sive pack­age on CMC that was sub­mit­ted in De­cem­ber 2020.

That tri­al en­rolled 10 pa­tients, and com­bined them with an­oth­er 15 al­ready treat­ed. Then, the 25 pa­tients will be com­pared with an­oth­er 25 pa­tients who “matched his­tor­i­cal con­trol pa­tients who have un­der­gone hematopoi­et­ic stem cell trans­plan­ta­tion.” Re­sults are ex­pect­ed from that in the sec­ond half of this year.

In a state­ment, CEO Manuel Litch­man said:

In light of our pos­i­tive ex­pe­ri­ence man­ag­ing the pri­or MB-107 CMC hold, and our abil­i­ty to se­cure FDA clear­ance to pro­ceed with that pro­gram, we be­lieve that our CMC team is well po­si­tioned to ad­dress the agency’s con­cerns around MB-207 once ad­di­tion­al clar­i­fi­ca­tion of the hold be­comes avail­able. Fur­ther­more, we re­main ful­ly com­mit­ted to the suc­cess of the piv­otal Phase II MB-207 clin­i­cal tri­al for chil­dren with XS­CID who have pre­vi­ous­ly re­ceived HSCT and re­quire re-treat­ment.

The news comes a lit­tle more than a month af­ter the com­pa­ny joined the NAS­DAQ. The stock hov­ered around the usu­al dol­lar-and-change mark Tues­day, as it sat around $1.17 per share around 11 a.m.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.