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.

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.

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The lat­est Cin­derel­la sto­ry in on­col­o­gy ends with a sud­den rout as up­dat­ed da­ta dis­play spooks in­vestors

NextCure’s turn as the Cinderella of cancer-focused biotechs was short-lived.
Just a few days after its shares $NXTC zoomed up more than 250% on some very early stage results in a SITC abstract, a more complete analysis over the weekend spiked the hype and left investors in high dudgeon as the stock price collapsed back towards earth Monday.
The focus at NextCure is centered on NC318, an antibody that is intended to shut down the immunosuppressive Siglec-15 — or S-15 — target. After adding a small group of patients to the readout, investigators circled 2 clinical responses, a complete and partial response, along with 4 stable disease cases in non-small cell lung cancer.

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Te­va spin­out rais­es $85M in IPO; No­var­tis beefs up gener­ics unit with $440M deal

→ After Teva spinout 89bio recently announced that its IPO was being held up, the company is back in the game offering 5,304,687 shares at a price of $16 per share. The company has raised $84.9 million IPO in gross proceeds and will be listed under the ticker symbol $ETNB. BofA Securities, SVB Leerink and RBC Capital Markets are the joint book-running managers for the offering. Oppenheimer & Co is the co-manager for the offering.
→ Looking to amp up its presence in Japan’s hospitals, Novartis has struck a deal to buy out Aspen’s portfolio of generics in the world’s third largest healthcare market. The pharma giant is paying $440 million for Aspen’s Japanese subsidiary.
→ Novartis said tropifexor, a non-bile acid FXR agonist, has scored on several key biomarkers of NASH in a Phase IIb trial, including reductions in hepatic fat, alanine aminotransferase and body weight compared to a placebo at 12 weeks.

Break­through sta­tus and promise of a speedy re­view ar­rives for Op­di­vo/Yer­voy com­bi­na­tion as Bris­tol-My­ers bites at Bay­er

Its frontline and single-agent aspirations have been set back, but Bristol-Myers Squibb just took a big step forward in its efforts to apply its checkpoint inhibitor Opdivo to liver cancer. The FDA has granted breakthrough status and priority review to a combination, second-line treatment.

The designation is for Opdivo (nivolumab) in combination with Yervoy (ipilimumab),  for treating advanced hepatocellular carcinoma (HCC), the most common form of liver cancer. The PD-L1 drug was already approved as a single-agent, second-line treatment for HCC. A PDUFA date was set for March 10, 2020 — just 4 months from now.

Third time un­lucky: Lipocine's lat­est quest to mar­ket their oral testos­terone drug snubbed again by FDA

Lipocine’s latest attempt at securing approval for its oral testosterone drug has fizzled yet again.

The Utah-based drug developer on Monday said the FDA has spurned its marketing application, indicating that some efficacy data on the drug, Tlando, was not up to scratch to treat male hypogonadism, a condition characterized by low production of the hormone testosterone, which is responsible for maintaining muscle bulk, bone growth, and sexual function.

UP­DAT­ED: De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.

As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.

Roche's SMA chal­lenge to Bio­gen's Spin­raza fran­chise looms larg­er with piv­otal win

Roche has just landed a crucial advance in scoring a come-from-behind win on the spinal muscular atrophy field, giving Novartis and Biogen a run for their money.

The update was brief, but Roche said risdiplam hit the primary endpoint in the placebo-controlled pivotal SUNFISH trial, meeting the threshold for change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment. The results, which is the second, confirmatory portion of a two-part study, involved 180 patients with type 2 or 3 spinal muscular atrophy between 2 and 25 years old.

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Roche steers Gazy­va in­to a new PhI­II pro­gram af­ter com­bo shows promise in lu­pus nephri­tis study

Roche is working on putting together a late-stage study for its monoclonal antibody Gazyva in patients with severe kidney disease associated with lupus after a combination approach helped patients in a mid-stage study.

The 125-patient NOBILITY trial evaluated Gazyva, combined with standard-of-care treatment mycophenolate mofetil or mycophenolic acid and corticosteroids, versus standard treatment alone. The combo met the main goal of inducing a statistically superior complete renal response (CRR) of 40% at week 76, versus 18% in patients given standard treatment, Roche said.