Bellerophon Hit As Late-Stage Trial Fails Following Interim Analysis

Shares of Bellerophon Therapeutics have plunged nearly 75 percent this morning after the company announced its Phase III pulmonary arterial hypertension (PAH) failed to meet endpoints following a pre-specified interim analysis from a Data Monitoring Committee.

Bellerophon said the late-stage PAH trial investigating INOpulse will be stopped for futility. The decision was based on the analysis of the first 75 patients enrolled in the INOvation-1 study after 16 weeks of treatment. The pre-specified analysis was conducted after half of the trial subjects completed 16 weeks of blinded treatment, Bellerophon said.

Warren, N.J.-based Bellerophon said trial data showed treatment with INOpulse sparked some improvement in pulmonary vascular resistance. However, the data monitoring committee deemed the primary endpoint of the trial, an overall change in the six-minute walk distance, was insufficient to support the continuation of the study.

Pulmonary arterial hypertension is high blood pressure in the lungs.  PAH occurs when the very small arteries throughout the lungs narrow in diameter, which increases the resistance to blood flow through the lungs. Over time, the increased blood pressure can damage the heart.

Fabian Tenenbaum, chief executive officer of Bellerophon, attempted to put a positive spin on things. He noted the company was disappointed in the early stoppage of the trial but said they were “encouraged” by the positive data in hemodynamics. Tenenbaum also said his team is pleased with the safety and tolerability profile of INOpulse.

“Over the next few weeks, we intend to further analyze the full dataset available to us from this interim analysis in order to determine the next steps in our PAH program,” Tenenbaum said in a statement.

Tenenbaum said the company remains positive about the potential of INOpulse, particularly given the results of the Phase II studies in pulmonary hypertension associated with interstitial lung disease (PH-ILD) and with chronic obstructive pulmonary disease (PH-COPD).

“Unlike PAH, patients with PH-ILD and PH-COPD have underlying lung disease for which systemic vasodilators have been ineffective and there are currently no approved therapies.  Our ongoing Phase IIb trial in PH-ILD is progressing well, with top-line results expected around the end of 2018.  PH-ILD is a significant unmet medical need which we believe represents a substantial potential market opportunity,” Tenenbaum added.

Bellerophon said it has sufficient financial resources to support its currently planned clinical R&D through the first half of 2019. Company stock plunged this morning after the announcement, sending prices well below $1 per share. Bellerophon, trading on the Nasdaq, closed at $2.28 per share on Monday. This morning, prices are down more than 75 percent to 53 cents per share, as of 10:11 a.m.

https://bit.ly/2OS7zaI

Express Scripts bars 48 drugs for 2019, hitting AbbVie, Gilead, Boehringer, more

It’s that time of year again—the time when lead PBM Express Scripts ignites pharma’s ire with a brand-new list (PDF) of formulary exclusions.

This time, Express Scripts barred 48 drugs from its national preferred formulary—22 of which compete against generics, 12 whose rivals use the same active ingredient at a lower net cost, and 11 that bear cheaper brand or biosimilar alternatives.

So who’s feeling the pain this time around? AbbVie, for one, whose pangenotypic hep C hotshot Mavyret was left off the list in favor of rivals from Merck & Co. and Gilead. The AbbVie drug has made waves in the hep C category, stealing share from its competitors and posting major growth numbers for the Illinois pharma.

But Gilead is mourning a couple losses of its own, including one-time hep C superstar Sovaldi and HIV treatment Atripla. On the HIV side, Express Scripts will favor other treatments from the Big Biotech, as well as products from nemesis GlaxoSmithKline and generics maker Mylan, in 2019.

Next-generation blood thinners Pradaxa from Boehringer Ingelheim and Savaysa from Daiichi Sankyo were also pushed out into the cold, leaving market-leaders Eliquis from Pfizer and Bristol-Myers Squibb and Xarelto from Johnson & Johnson more room to to duke it out at the top of the class.

To hear Express Scripts tell it, drugmakers asked for the new round of exclusions with their pricing antics, leaving the PBM no choice but to keep their drugs off the “covered” list.

“Despite promises to limit price increases, drugmakers are trying to game the market by delaying generic competition, blocking access to safe and effective biosimilars, and coyly deferring—not cancelling—list-price increases,” Express Scripts said on its website, referencing a recent Trump-appeasing move from Pfizer.

“This is why our work to expand access and maximize value is more important now than ever,” Express Scripts added.

What it didn’t mention, though, is that refusing to cover pricey new therapies also helps its own bottom line, one fact that’s led pharmas to point the finger at payers in attempts to deflect the public’s drug-pricing criticism.

https://bit.ly/2Mc8eoO

PTC Therapeutics reports Q2 EPS (21c), consensus (29c)

Reports Q2 revenue $68.7M, consensus $65.7M.

https://bit.ly/2MsU2Vs

Cara Therapeutics reports Q2 EPS (52c), consensus (32c)

Reports Q2 revenue $2.87M, consensus $6.47M.

https://bit.ly/2OSepgj

Voyager Therapeutics reports Q2 EPS (80c), consensus (67c)

Reports Q2 revenue $2.6M, consensus $2.8M. “The significant progress made during the second quarter with our lead program VY-AADC for Parkinson’s disease and our pipeline programs positions Voyager to potentially achieve important milestones for the remainder of this year and into 2019. These include enrolling our pivotal program for VY-AADC for Parkinson’s disease and providing longer-term results from the Phase 1 trials as well as presenting preclinical data on our programs targeting ALS SOD-1 and Huntington’s disease. In addition, we continue to advance our collaboration with AbbVie targeting vectorized monoclonal antibodies directed against tau for the treatment of Alzheimer’s disease and other neurodegenerative diseases,” said CEO Andre Turenne.

https://bit.ly/2OjFeZP

Zafgen reports Q2 EPS (57c), consensus (59c)

“2018 is an important year of execution on our plan for our novel second-generation MetAP2 pipeline,” said Jeffrey Hatfield, Chief Executive Officer. “We’ve continued to make significant progress this year, highlighted by positive data from the initial part of our Phase 2 clinical trial of ZGN-1061 in patients with type 2 diabetes, launch of the natural history study of PWS, advancement towards clinical testing for ZGN-1258, strengthening of our balance sheet, and key personnel additions. We expect this progress to continue throughout the remainder of 2018 and beyond, with near-term milestones for all of our key pipeline programs.” As of June 30, 2018, the Company had cash, cash equivalents and marketable securities totaling $75.8 million. In July 2018, the Company completed an underwritten public offering of its common stock, which included the full exercise of the underwriters’ option to purchase additional shares, resulting in net proceeds of approximately $64.6 million. After giving effect to the estimated net proceeds from the Company’s public offering of common stock, the unaudited pro forma cash, cash equivalents and marketable securities balance was $140.4 million as of June 30, 2018. The Company expects that its cash, cash equivalents and marketable securities balance will be greater than $100 million as of December 31, 2018.

https://bit.ly/2niR4YD

American Renal Associates reports Q2 EPS 20c, consensus 17c

Reports Q2 revenue $217.2M, consensus $204.01M. Adjusted EBITDA less noncontrolling interests was $31.5 million as compared to $27.4 million in Q2 2017; Total dialysis treatments increased 5.6%, of which 4.5% was non-acquired growth. Normalized total treatment growth was 6.3% and normalized non-acquired growth was 5.3%.

https://bit.ly/2OkojWS