The Sen­ate fum­bles its first stab at phar­ma prof­i­teer­ing, keep­ing the is­sue front and cen­ter in 2017

End­points News as­sess­es the big bio­phar­ma R&D sto­ry this week in a spe­cial year-end send­off. Chances are, this is al­so what we’ll be talk­ing about in 2017. And it will cer­tain­ly be front and cen­ter at the up­com­ing J.P. Mor­gan Health­care Con­fer­ence.

The Sen­ate of­fers a weak re­sponse to price goug­ing

The Sen­ate this week high­light­ed just how lit­tle the bio­phar­ma in­dus­try needs to fear from law­mak­ers when it comes to con­trol­ling drug prices right now. In­ves­ti­gat­ing four clear cas­es of price goug­ing on old prod­ucts, two or­ches­trat­ed by Mar­tin Shkre­li, the Sen­ate pol­ished off a mix of dusty reg­u­la­to­ry in­cen­tives as their sug­ges­tion for rein­ing in prof­i­teer­ing: “spe­cial” pri­or­i­ty re­views when need­ed, vouch­ers to help re­ward a fast re­sponse, maybe a spe­cial reim­por­ta­tion pass to pro­vide cheap com­pe­ti­tion, a man­date to move gener­ics through the FDA faster. None of it would ac­tu­al­ly nip the whole thing in the bud. “It is pos­si­ble that the busi­ness mod­el pur­sued by the Valeants and Tur­ings of the world was at­trac­tive in part be­cause it was le­gal,” the re­port states, in a mas­sive un­der­state­ment. (Ya think?) Noth­ing the sen­a­tors sug­gest­ed would ac­tu­al­ly pre­vent the next Shkre­li from do­ing ex­act­ly the same thing. If law­mak­ers are un­will­ing to tack­le ob­vi­ous price goug­ing, then what are the chances they can ever come to grips with the much big­ger is­sue of sys­temic rip-offs, the an­nu­al price spikes that have kept Big Phar­ma look­ing healthy as their R&D ef­forts con­tin­ue to un­der­per­form?

It’s time to get re­al

Drug pric­ing as a po­tent po­lit­i­cal is­sue, though, isn’t go­ing any­where. As Shkre­li told mein a Twit­ter ex­change on Thurs­day, the two deals he en­gi­neered for Tur­ing and Retrophin are still mak­ing plen­ty of “guap” as law­mak­ers fud­dle about. Don­ald Trump has made his own broad, un­de­fined pledge to con­trol pric­ing. And elect­ed of­fi­cials have to be acute­ly aware that the pub­lic’s anger will on­ly grow. One of the most com­pelling in­dus­try re­spons­es has been Al­ler­gan CEO Brent Saun­ders’ pledge to hold an­nu­al price in­creas­es to sin­gle dig­its.

Fair pric­ing prac­tices on old­er drugs will be es­sen­tial to main­tain­ing greater con­trol over the price of new drugs. It’s im­por­tant to re­mem­ber that pub­lic anger is not fixed on the price of im­por­tant new drugs that make a dif­fer­ence in peo­ple’s lives. It’s the old­er drugs, where the price goes up when the fran­chise should be fad­ing in val­ue, that rile the peo­ple and of­fend their sense of fair­ness. No one likes be­ing ripped off. And with­out ag­gres­sive pric­ing on many, though not nec­es­sar­i­ly all, new drugs, R&D will be­come un­sus­tain­able.

If this is­sue has your at­ten­tion, I’d en­cour­age you to come to End­points News’ first live event dur­ing the J.P. Mor­gan Health­care Con­fer­ence in San Fran­cis­co, where we’ll be joined by Saun­ders and a great pan­el dis­cussing the sin­gle most press­ing is­sue of the year.

Your tick­et rev­enue will be used to help grow the pub­li­ca­tion in 2017. So you can view this as a way to par­tic­i­pate in a great dis­cus­sion, a net­work­ing event you won’t want to miss, and a sign of your sup­port for what we’re do­ing here.

John Car­roll, Ed­i­tor

Hap­py hol­i­days, and I hope to see you in San Fran­cis­co Jan­u­ary 10.— John Car­roll

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

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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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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See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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Hugo Peris, Spiral Therapeutics CEO

Hear­ing-fo­cused biotech grabs trio of pro­grams from Oton­o­my's fire sale

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.