Is bio­phar­ma’s first $1.5M ther­a­py now in Phase III?

Key­ing off of Spark Ther­a­peu­tics’ roll­out of Lux­tur­na as the first ap­proved gene ther­a­py in the US, the an­a­lysts at Leerink have been crunch­ing the num­bers on what the first he­mo­phil­ia gene ther­a­pies could fetch. And by rais­ing their es­ti­mate to $1.5 mil­lion, they may well be tempt­ing fate at a time the de­bate over drug pric­ing is reach­ing the boil­ing point in the US.

Spark sat­is­fied most an­a­lysts with its $425,000 price per eye for Lux­tur­na. And Leerink says that the pos­i­tive re­sponse by Har­vard Pil­grim to a 30-month dura­bil­i­ty start­ing point for a gene ther­a­py pric­ing agree­ment set the stage for many of the rest of the field to fol­low suit. And that will jus­ti­fy a $1.5 mil­lion-plus price for a ther­a­py that could re­place cur­rent stan­dards in he­mo­phil­ia with a sim­i­lar track record on dura­bil­i­ty.

Af­ter check­ing with some KOLs in the field, Leerink of­fered:

As a re­sult of this work, we are rais­ing our as­sumed price for he­mo­phil­ia gene ther­a­pies in our cov­er­age uni­verse to $1.5M per pa­tient in the US ($1M in the EU), an in­crease from pri­or as­sump­tions of $350,000-$500,000 (he­mo­phil­ia A) and $500,000 (he­mo­phil­ia B) per pa­tient. Our checks in­di­cate that there may be some head­room above this price, po­ten­tial­ly up to $2M per pa­tient on a gross ba­sis, but giv­en the un­cer­tain­ty about re­spon­sive­ness, re­bates and oth­er fac­tors, we be­lieve $1.5M is a jus­ti­fi­able es­ti­mate for net price per pa­tient at this junc­ture….

We be­lieve the man­u­fac­tur­ers are test­ing the wa­ters cur­rent­ly as they em­bark on Phase 3 tri­als in or­der to de­ter­mine where pay­ors push back sig­nif­i­cant­ly. Based on our checks, a price some­where in the range of $1-2M per pa­tient seems most like­ly.

The big rea­son for that 7-fig­ure price tag is based on pay­ers’ will­ing­ness to cov­er the $580,000 to $800,000 price per pa­tient for fac­tor re­place­ment ther­a­py, giv­ing them some up­side even with a $1 mil­lion-plus stick­er for a once-and-done gene ther­a­py. And that could have a pro­found ef­fect on Spark $ONCE, Bio­Marin $BM­RN and uniQure $QURE, which are all en­gaged in de­vel­op­ment ef­forts.

As long as the av­er­age pa­tient can re­ly on fac­tor re­place­ment ther­a­py, Leerink adds, the low hang­ing fruit for the de­vel­op­ers ear­ly on will be among the most se­vere pa­tients, and stick­ing with that small pa­tient pop­u­la­tion al­so helps jus­ti­fy the big num­ber.

At this stage of the game, the biotechs lead­ing this field will like­ly hedge their bets, steer­ing clear of of­fer­ing any num­bers they may have in mind as they test the wa­ters on the fi­nal price tag. But Leerink just gave them a big op­por­tu­ni­ty to con­tin­ue their pri­vate pay­er talks with some big num­bers in mind.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.