Lib­er­tar­i­an ideas on FDA dereg­u­la­tion threat­en biotech; It's time for some re­al pow­er to re­form drug prices, rea­son­ably

Do you sin­cere­ly want to dereg­u­late drug de­vel­op­ment?

Sil­i­con Val­ley’s Bal­a­ji Srini­vasan had quite a habit of tack­ling the FDA on Twit­ter, crit­i­ciz­ing the agency for its slow and back­ward ways. But all that came back to haunt him af­ter re­porters picked up on the news that he and fel­low ‘seast­ead­er’ Jim O’Neill had been in to chat with Don­ald Trump about top jobs at the FDA. Sud­den­ly, that Twit­ter chan­nel van­ished in­to thin air, as we re­port­ed first. But his Tweets were saved, and live on, even as we hear that Srini­vasan may not be in line for that job any more. Trump’s fi­nal pick to run the FDA will have a huge in­flu­ence on how the agency plans to speed drug de­vel­op­ment. And we have a right and a need to suss things out for our­selves. So far, this episode ranks as an­oth­er rea­son to sus­pect that the FDA may soon be head­ed for a wrong turn, es­pe­cial­ly if the new fo­cus is on a sub­stan­tial dereg­u­la­tion of drug de­vel­op­ment born out of a dis­gust for the bu­reau­cra­cy.

Medicare price ne­go­ti­a­tions can ben­e­fit bio­phar­ma too.

Speak­ing of Trump, our new pres­i­dent got the chance to clar­i­fy his think­ing re­gard­ing drug prices in a week­end in­ter­view with The Wash­ing­ton Post. As we sus­pect­ed, his main weapon in the war against list prices will be Medicare ne­go­ti­a­tions — now banned by in­dus­try-friend­ly law­mak­ers. The in­dus­try des­per­ate­ly doesn’t want to see this, but in re­al­i­ty it makes for an ef­fec­tive way to get ac­tu­al dis­count prices on dis­play for all to see. The Amer­i­can pub­lic has a right to see what’s ac­tu­al­ly be­ing paid for ther­a­peu­tics, and this is one way to set a base­line dis­count price that is read­i­ly trans­par­ent. The free ride on ever-ris­ing drug prices is over, and we all know it. The soon­er this hap­pens, the bet­ter for every­one. If a price can’t sur­vive the light of day, it’s too high. In the mean­time, the in­dus­try can move more to­ward a per­for­mance-based pric­ing method.

Mallinck­rodt joins the price goug­ing club, and gets away with “mur­der.”

One of the rea­sons why drug prices are so con­tro­ver­sial is that the fed­er­al gov­ern­ment is vir­tu­al­ly help­less when it comes to pre­vent­ing a com­pa­ny from jack­ing up prices as­tro­nom­i­cal­ly overnight. Quest­cor proved that more than three years ago, when it brazen­ly bought a ri­val to Ac­thar to pre­vent com­pe­ti­tion. And Mallinck­rodt bought in to that rigged game by buy­ing Quest­cor. This week, Mallinck­rodt was cit­ed for il­le­gal­ly block­ing com­pe­ti­tion, but got away with on­ly a $100 mil­lion fine. The com­pa­ny paid for that with the in­creased rev­enue it earned on Ac­thar in just 2015, as its price con­tin­ued to soar. This is what Trump means when he says phar­ma is get­ting away with mur­der. It may take years for a ri­val to get ap­proved and on the mar­ket. And Mallinck­rodt is still free to jack prices. This has to end with some kind of ef­fec­tive le­gal mech­a­nism to pre­vent abus­es. Your next Mar­tin Shkre­li or Mallinck­rodt is sit­ting just around the cor­ner. And the longer we wait, the more like­ly it is that re­form will overzeal­ous­ly dam­age the way in­no­va­tion is fi­nanced in bio­phar­ma.

Our lat­est lists on ven­ture cash un­der­score sol­id fun­da­men­tals for biotech. Let’s not screw it up now.

Biotech is de­pen­dent on the flow of bil­lions of dol­lars in ven­ture cash. And we love noth­ing quite as much as track­ing where the mon­ey is com­ing from as well as what re­gions it is go­ing to. Al­ways enor­mous­ly pop­u­lar with read­ers, this year’s sto­ries paint a sim­ple tale of the dom­i­nant role played by two cru­cial hubs — Boston/Cam­bridge and the Bay Area — as well as a group of savvy in­vestors who are in­stru­men­tal in fos­ter­ing and fund­ing new in­no­va­tion in drug de­vel­op­ment. That ma­chine has been hum­ming along nice­ly now for sev­er­al years, point­ing to the fact that the fun­da­men­tals in pri­vate biotech are strong and durable. That bodes well for the years ahead, as ma­jor re­forms are be­ing planned, which we should all ap­pre­ci­ate. Re­form­ers might like to keep that in mind.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.

FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,300+ biopharma pros reading Endpoints daily — and it's free.