Re­genxbio scores $180M vec­tor deal; Bris­tol-My­ers ex­pands agree­ment to test Op­di­vo with In­fin­i­ty's IPI-549

Abeona Ther­a­peu­tics $ABEO has li­censed Re­genxbio’s $RGNX NAV AAV9 vec­tor for four dis­eases: San­fil­ip­po syn­drome type A (MPS II­IA), San­fil­ip­po syn­drome type B (MPS II­IB), In­fan­tile Bat­ten Dis­ease, al­so known as neu­ronal ceroid lipo­fus­ci­nosis type 1 (CLN1 Dis­ease), and Ju­ve­nile Bat­ten Dis­ease, al­so known as neu­ronal ceroid lipo­fus­ci­nosis type 3 (CLN3 Dis­ease). Re­genxbio is guar­an­teed $40 mil­lion in cash for the deal, in­clud­ing $20 mil­lion in near-term pay­ments, with an­oth­er $140 mil­lion in po­ten­tial fees and mile­stones.

With six ap­proved PD-1/L1 drugs on the mar­ket and a pletho­ra of oth­ers in de­vel­op­ment, mak­ers of check­point in­hibitors con­tin­ue to test their of­fer­ings in com­bi­na­tion with oth­er drugs to eke out a big­ger slice of the lu­cra­tive on­col­o­gy mar­ket. On Mon­day, Bris­tol-My­ers $BMY ex­pand­ed its agree­ment with In­fin­i­ty Phar­ma­ceu­ti­cals’ $IN­FI to test Op­di­vo in com­bi­na­tion with In­fin­i­ty’s PI3K-gam­ma in­hibitor, IPI-549, in pa­tients with ad­vanced urothe­lial can­cer. The Phase II tri­al is ex­pect­ed to be­gin in the first half of 2019. A com­bi­na­tion of Op­di­vo and IPI-549 con­tin­ues to be eval­u­at­ed in a Phase I study in pa­tients with ad­vanced sol­id tu­mors.

In oth­er can­cer-re­lat­ed news, Take­da has ex­er­cised its ex­clu­sive op­tion to li­cense Crescen­do Bi­o­log­ics’ hum­a­bod­ies for one of its on­col­o­gy tar­gets, in a move that comes sub­stan­tial­ly ear­li­er than planned, Crescen­do said on Mon­day. The mul­ti-tar­get col­lab­o­ra­tion and li­cense agree­ment was set up in 2016. Crescen­do’s hum­a­body-based prod­ucts are an in­ves­ti­ga­tion­al class of pro­tein ther­a­peu­tics, which are de­signed to pen­e­trate and ac­cu­mu­late in tis­sue/tu­mours whilst clear­ing quick­ly from cir­cu­la­tion to min­imise sys­temic tox­i­c­i­ty.

In a sep­a­rate on­col­o­gy tie-up, lit­tle MEI Phar­ma $MEIP has grant­ed Tokyo-list­ed Ky­owa Hakko Kirin ex­clu­sive rights to de­vel­op and com­mer­cial­ize its ex­per­i­men­tal drug ME-401 in Japan for a $10 mil­lion up­front and po­ten­tial mile­stone pay­ments of up to $87.5 mil­lion. The drug, a PI3K delta in­hibitor, is be­ing de­vel­oped by MEI to treat B-cell ma­lig­nan­cies, and MEI is plan­ning to ini­ti­ate a Phase II study to eval­u­ate the drug in pa­tients with fol­lic­u­lar lym­phoma.

Ger­many’s Grü­nen­thal is now the sole own­er of the der­mal pain patch Quten­za af­ter it ac­quired the re­main­ing rights to the prod­uct from Acor­da Ther­a­peu­tics $ACOR. Back in 2016, Grü­nen­thal ac­quired ex­clu­sive com­mer­cial rights for the patch — which con­tains cap­saicin, the ac­tive in­gre­di­ent in chili pep­pers — in Eu­rope, Mid­dle East and Africa. Grü­nen­thal has made a se­ries of in­vest­ments to ex­pand its ar­se­nal of pain prod­ucts, in­clud­ing ac­quir­ing the Eu­ro­pean rights to Nex­i­um and the glob­al (ex US and Japan) rights to Vi­mo­vo

An­oth­er Ger­man com­pa­ny is al­so in the news. The Mainz-based biotech BioN­Tech has tied up with the Uni­ver­si­ty of Penn­syl­va­nia to de­vel­op mR­NA vac­cines to pre­vent and treat a host of in­fec­tious dis­eases. The com­pa­ny, which al­ready has part­ner­ships with with Roche’s Genen­tech, Pfiz­er, Gen­mab, Eli Lil­ly, Sanofi and Bay­er, is bet­ting that mR­NA-based vac­cines rep­re­sent an op­por­tu­ni­ty to sur­pass con­ven­tion­al vac­cine ap­proach­es be­cause of their ca­pac­i­ty for rapid de­vel­op­ment and their po­ten­tial for low-cost man­u­fac­tur­ing and po­ten­tial­ly safe ad­min­is­tra­tion.

Is­rael’s Mapi Phar­ma has picked up its sec­ond $10 mil­lion eq­ui­ty in­vest­ment from Chi­na’s Zhe­jiang Jingx­in Phar­ma­ceu­ti­cal. The Chi­nese CNS drug­mak­er made its ini­tial in­vest­ment in Mapi back in 2016.

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.”

EU re­port­ed­ly eye­ing vac­cines deal for mon­key­pox out­break; Nas­daq tells Mereo to get stock price up or face delist­ing

The EU is reportedly working toward a centralized procurement deal for smallpox vaccines to tackle the rising number of cases of monkeypox, according to sources who spoke to the Financial Times.

Andrea Ammon, director of Europe’s CDC, told the FT that the European Commission is “definitely looking at what can be done centrally” and central procurement is “one of the options that are looked at but of course countries are also” looking into bilateral avenues.

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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