Thumbs up: Can­cer drug R&D is ex­plod­ing, get ready for the pric­ing de­bate to fol­low

I ar­rived in Chica­go last night, still try­ing to sort out how to pri­or­i­tize the news com­ing out of AS­CO this week­end. In the lead up, I had a chance to do a pair of sto­ries that looked at new re­ports cov­er­ing the rapid pro­lif­er­a­tion of PD-(L)1 com­bos along with a 7.7% one-year growth rate in late-stage can­cer drug stud­ies, com­ing in at 631 piv­otal-stage ef­forts.

This didn’t hap­pen by ac­ci­dent.

Over the last few years we’ve seen a tru­ly rev­o­lu­tion­ary change in the way on­col­o­gy drugs are de­vel­oped. Backed in par­tic­u­lar by the FDA’s break­through pro­gram, now lib­er­al­ly ap­plied along with fast-track sta­tus and pri­or­i­ty re­views, the bio­phar­ma in­dus­try is com­pet­ing like nev­er be­fore to carve out new ter­ri­to­ry in can­cer treat­ment.

I well re­mem­ber a lot of the ini­tial hoot­ing from some an­a­lysts at the idea that the FDA’s BTD pro­gram would make a dif­fer­ence. But no one is hoot­ing now. The agency — and in par­tic­u­lar Richard Paz­dur — can­did­ly em­braced an open-door ap­proach on can­cer R&D. And that’s the path FDA com­mis­sion­er Scott Got­tlieb wants the rest of the FDA di­vi­sions to fol­low.

Richard Paz­dur

When a bu­reau­cra­cy sets new goals, things change. So I’ll be ex­pect­ing lots of fresh ac­tion on the BTD front over the next few years, for all kinds of dis­eases.

There are bil­lions of dol­lars at stake here, which is as it should be. The greater the pay­back, the greater the R&D in­vest­ment and the greater the re­wards for pa­tients, who are see­ing some im­por­tant and some­times dra­mat­ic im­prove­ment in the way many can­cers are treat­ed.

Out of this rev­o­lu­tion, though, will come plen­ty of added pres­sure on drug mak­ers to find a way to rea­son­ably price these new drugs and the com­bos com­ing down the pipeline. Af­ter get­ting side­lined at the Trump ad­min­is­tra­tion, changes on pub­lic pol­i­cy around drug pric­ing may well get lost in all the shout­ing in DC these days.

But you can be sure that pay­ers haven’t lost their fo­cus, es­pe­cial­ly as six-fig­ure com­bos start to make the rounds. So the in­dus­try will need to have a more co­her­ent ap­proach to pay for per­for­mance.

On­col­o­gy now rep­re­sents the po­ten­tial that drug R&D has in sig­nif­i­cant­ly im­prov­ing the stan­dard of care in a short pe­ri­od of time. Next, it has to lead the way on mar­ket-based so­lu­tions to the pric­ing de­bate.

That next part could prove to be much, much more chal­leng­ing.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Cy­to­ki­net­ics’ ALS drug fails PhI­II, leav­ing the biotech with a sin­gle late-stage prospect

Cytokinetics’ candidate for the muscle disease amyotrophic lateral sclerosis, or ALS, failed a Phase III trial, the Bay Area biotech announced Friday morning.

At a second interim analysis of the trial, an independent review committee recommended that Cytokinetics discontinue its COURAGE-ALS trial for reldesemtiv, as it “found no evidence of effect” compared to placebo on the primary or key secondary endpoints.

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CHMP gives thumbs-up for We­govy use in ado­les­cents, along with nine new drug rec­om­men­da­tions

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended nine drugs for approval this week while also giving thumbs up for six expanded indications, including Novo Nordisk’s approved obesity medication Wegovy for younger people. Wegovy is already approved as an obesity treatment in the EU for adults, and the new indication would allow prescriptions for adolescents aged 12 and older.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Green­Light re­ceives buy­out of­fer; Apol­lomics com­pletes SPAC merg­er

RNA biotech GreenLight Biosciences has been handed an offer for potential acquisition.

GreenLight said in a release that it has received a non-binding “indication of interest” from Fall Line Endurance Fund to acquire GreenLight’s capital stock for $0.60 per share in cash. The release said any potential agreement between the two parties would depend on certain conditions.

Through a special committee, the biotech will evaluate the offer but added there’s no certainty a deal will go forward. GreenLight will also not make any more announcements until a deal comes through or “otherwise determines” a statement is necessary.

TScan Therapeutics' departing CEO David Southwell and CSO/COO Gavin MacBeath

TCR up­start an­nounces CEO ex­it, with CSO now act­ing re­place­ment

A public T cell biotech’s chief executive has decided to leave the company.

TScan Therapeutics said Friday morning that CEO David Southwell stepped down earlier this week, leaving both his chief executive and board member roles. Filling in is Gavin MacBeath, the company’s CSO and COO. He became the acting CEO on Tuesday, and will continue to remain CSO and COO, TScan’s announcement read.

Sar­to­rius to ac­quire French man­u­fac­tur­er for $2.6B+ in cell and gene ther­a­py play

The German life science group Sartorius will be picking up French contract manufacturer Polyplus for the price of €2.4 billion, or $2.6 billion.

On Friday, Sartorius announced the acquisition through its French subgroup, Sartorius Stedim Biotech, which will be acquiring Polyplus from private investors ARCHIMED and WP GG Holdings IV. Polyplus has 270 employees and produces materials and components that go into making viral vectors that are used in cell and gene therapies. This includes DNA/RNA reagents as well as plasmid DNA. Polyplus has locations in France, Belgium, China and the US.

Austin biotech Mol­e­c­u­lar Tem­plates lays off more than 100 staffers as pipeline nar­rows

Molecular Templates is ridding itself of a Phase I HER2 asset and fine-tuning its pipeline to focus on three programs and a preclinical Bristol Myers Squibb collaboration. With the narrowed scope on its so-called engineered toxin bodies, the Austin, TX biotech is laying off about half of its staff.

That’s a little more than 100 employees, per an SEC filing. Molecular’s layoffs, approved by its board Wednesday, add to the dozens of pullbacks in the industry in the first three months of 2023.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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