Puma touts its lat­est ad­vances in a bas­ket tri­al for Ner­l­ynx, as Eu­ro­peans ap­pear ready to re­ject the drug

While Eu­ro­pean reg­u­la­tors ap­pear ready to slap down Puma Biotech­nol­o­gy’s $PBYI first shot at an ap­proval for Ner­l­ynx (ner­a­tinib), the com­pa­ny is push­ing ahead with a bas­ket tri­al it hopes to use to even­tu­al­ly use to broad­en its ini­tial FDA ap­proval for breast can­cer to a whole range of sol­id tu­mors.

In the first cut of the SUM­MIT study, re­searchers say they not­ed that “re­spons­es were ob­served in pa­tients with breast, cer­vi­cal, bil­iary, sali­vary and non-small-cell lung can­cers, which led to co­hort ex­pan­sions in these tu­mor types. No ac­tiv­i­ty was ob­served in the HER3-mu­tant co­hort.”

Alan Auer­bach

Rather than mount an in­di­vid­ual study on each can­cer type, Puma is pur­su­ing a bas­ket de­sign that groups to­geth­er pa­tients with dif­fer­ent types of can­cers to see how their drug can be more rapid­ly ac­cel­er­at­ed to each, or where they should avoid go­ing.

“SUM­MIT is al­so sig­nif­i­cant in that it will pro­vide the largest body of clin­i­cal da­ta to date on the use of an ir­re­versible pan-HER in­hibitor in pa­tients who have sol­id tu­mors with so­mat­ic HER2 or HER3 mu­ta­tions,” says CEO Alan Auer­bach.

The CHMP, though, has raised con­cerns about the clin­i­cal rel­e­vance of the da­ta Puma gath­ered in their first Phase III study.

Ner­l­ynx is al­so linked to a high rate of se­ri­ous di­ar­rhea, which didn’t pre­vent a lop­sided vote in its fa­vor by a group of ex­pert FDA ad­vis­ers, who ap­peared sat­is­fied that the side ef­fect could usu­al­ly be con­trolled with dai­ly dos­es of lop­eramide.

Michael Schmidt

Even so, in the SUM­MIT tri­al “four pa­tients (2.8%) per­ma­nent­ly dis­con­tin­ued ner­a­tinib and 21 pa­tients (14.9%) had dose in­ter­rup­tions due to di­ar­rhea.”

In the mean­time, an­a­lysts are watch­ing for a new round of Phase III da­ta that should be com­ing up soon. In a re­cent note, Leerink’s Michael Schmidt said:

This sec­ond piv­otal Phase III tri­al of ner­a­tinib in the 3rd line metasta­t­ic HER2+ breast can­cer set­ting com­plet­ed pa­tient ac­cru­al in mid-17. Top-line re­sults are ex­pect­ed in 1H18. NALA is a dou­ble-blind con­trolled tri­al com­par­ing Ner­l­ynx com­bined with capecitabine (Xelo­da) vs. Tykerb (la­p­a­tinib) com­bined with Xelo­da in 600 pa­tients. The tri­al is con­duct­ed un­der an SPA agree­ment with the FDA with the co-pri­ma­ry end­points be­ing PFS and OS. PBYI plans to sub­mit the NDA and MAA based on the PFS re­sults – if pos­i­tive.

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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Deborah Dunsire. Lundbeck

Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation for a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month.

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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.