AKAO Slumps On FDA Panel Vote, ESPR Awaits Data In May, STAA Shines

(RTTNews) - Today's Daily Dose brings you news about FDA panel's vote on Achaogen's Plazomicin; Karyopharm's public offering; Cellectis' progress in TALEN gene-edited product pipeline; Globus Medical's Q1 financial results; a federal Court's ruling, which has gone in favor of Progenics/Valeant, and Esperion's pivotal phase III long-term safety study results of Bempedoic acid.

Read on…

Shares of Achaogen Inc. (AKAO) slumped over 28% in extended trading on Wednesday, after the FDA panel delivered its verdict on the Company's investigational treatment Plazomicin.

The Company is seeking approval of Plazomicin IV for the treatment of complicated urinary tract infections (cUTI), including pyelonephritis, and bloodstream infections (BSI) due to certain Enterobacteriaceae in patients who have limited or no alternative treatment option.

While the FDA panel members unanimously backed Plazomicin for the treatment of complicated urinary tract infections, they voted 4-11 against recommending approval of the drug candidate for the treatment of bloodstream infections in patients with limited or no treatment options.

The FDA's final decision on Plazomicin is expected on June 25, 2018.

AKAO closed Wednesday's trading at $14.70, up 2.73%. In after-hours, the stock was down 28.91% to $10.45.

Cellectis (CLLS) has filed an Investigational New Drug application to the FDA, seeking approval to initiate a Phase 1 clinical trial for UCART22, the Company's second wholly controlled TALEN gene-edited product candidate, for the treatment of B-cell acute lymphoblastic leukemia (B-ALL) in adult patients.

If all goes well as planned, the Company plans to initiate a Phase I clinical trial of UCART22 in the third quarter of 2018.

Another wholly controlled TALEN gene-edited product candidate of Cellectis is UCART123, under phase I trial in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).

Cellectis' UCART19, which is under phase I trial in patients with acute lymphoblastic leukemia, is also a TALEN gene-edited product candidate, but is being co-developed by Servier and Pfizer.

CLLS closed Wednesday's trading at $28.86, up 2.38%. In after-hours, the stock was up 2.22% to $29.50.

Shares of Esperion Therapeutics Inc. (ESPR) slumped more than 35% on Wednesday, despite the Company reporting positive top-line results from its pivotal phase III long-term safety study of Bempedoic acid in high-risk patients with atherosclerotic cardiovascular disease (ASCVD) who are inadequately controlled with current lipid-modifying therapies.

The study, dubbed Study 1 or 1002-040, which included 2,230 patients, met the primary endpoint of safety and tolerability, and the key efficacy endpoint with on-treatment LDL-C lowering of an additional 20 percent at twelve weeks in the intent to treat analysis.

Patients treated with bempedoic acid also achieved a significant reduction of 22 percent in high-sensitivity C-reactive protein (hsCRP), an important marker of the underlying inflammation associated with cardiovascular disease.

However, the number of fatal adverse events in the treatment group spooked investors - there were 13 deaths in the treatment group compared to 2 in placebo. The fatal adverse events were unrelated to the study drug, says the Company.

Upcoming milestones:

-- Top-line results from the pivotal Phase 3 Study 3 (1002-046) of bempedoic acid in ASCVD patients on background therapy of less than approved daily starting doses of statins expected in May. -- Top-line results from the pivotal Phase 3 Study (1002FDC-053) of the bempedoic acid / ezetimibe combination pill in ASCVD patients on maximally tolerated statin background therapy expected in August 2018. -- Top-line results from the pivotal Phase 3 Study 2 (1002-047) of bempedoic acid in ASCVD patients on maximally tolerated statin background therapy expected in September 2018.

ESPR closed Wednesday's trading at $45.75, down 35.11%.

Globus Medical Inc. (GMED) has reported strong results for the first quarter of 2018, and has increased its full-year forecast.

On a non GAAP basis, net income for the first quarter of 2018 rose to $39.54 million or $0.39 per share from $28.71 million or $0.30 per share in the year-ago quarter. Worldwide sales were $174.4 million in the recent first quarter, up from $155.8 million in the first quarter of 2017.

Analysts polled by Thomson Reuters were expecting EPS of $0.36 on revenue of $167.89 million.

The company has also increased guidance for full year 2018 sales to $695 million from $690 million and non-GAAP fully diluted earnings per share to $1.52 from $1.50.

GMED closed Wednesday's trading at $49.65, down 0.04%.

Karyopharm Therapeutics Inc. (KPTI) has offered to sell 9.15 million shares of its common stock to the public at a price of $14.75 each.

The underwriters have been given a 30-day option to purchase up to 1.37 million additional shares of Company's common stock. The gross proceeds to Karyopharm from the offering are expected to be $135.0 million. The offering is slated to close on or about May 7, 2018, subject to customary closing conditions.

On April 30, 2018, the Company reported positive top-line data from Part 2 of its phase IIb trial of Selinexor in patients with Penta-Refractory Multiple Myeloma, dubbed STORM.

Near-term Catalysts:

-- Karyopharm plans to submit a New Drug Application to the FDA during the second half of 2018, with a request for accelerated approval for oral Selinexor as a new treatment for patients with penta-refractory multiple myeloma. -- The Company also plans to submit a Marketing Authorization Application to the European Medicines Agency in early 2019 with a request for conditional approval. -- A phase III study evaluating Selinexor in combination with Velcade and dexamethasone (SVd) for the treatment of patients with multiple myeloma who have had one to three prior lines of therapy, dubbed BOSTON, is ongoing - with top-line data anticipated in 2019.

KPTI closed Wednesday's trading at $15.52, up 6.96%.

Neurocrine Biosciences Inc. (NBIX) is scheduled to present new data from RE-KINECT, the largest real-world screening study to date of patients with clinician confirmed possible tardive dyskinesia on May 8, 2018.

The RE-KINECT study is designed to better understand the potentially broad reaching impact of symptoms of possible tardive dyskinesia patients treated with antipsychotic medications.

The Company will also present new data from the KINECT 4 study on long-term treatment with INGREZZA capsules in participants with schizophrenia/schizoaffective disorder or mood disorder on May 8, 2018.

INGREZZA is the first FDA approved treatment for adults with tardive dyskinesia. It was approved last April.

NBIX closed Wednesday's trading at $80.08, down 5.11%.

Progenics Pharmaceuticals Inc. (PGNX) and partner Valeant Pharmaceuticals International Inc. (VRX) have a reason to cheer as a federal Court's ruling pronounced on Wednesday prevents generic competition for RELISTOR injection in the United States until 2024.

Progenics has exclusively licensed development and commercialization rights for RELISTOR, to Valeant. RELISTOR Tablets (450 mg once daily) are approved in the United States for the treatment of opioid-induced constipation (OIC) in patients with chronic non-cancer pain. RELISTOR Subcutaneous Injection (12 mg and 8 mg) is a treatment for OIC approved in the United States and worldwide for patients with advanced illness and chronic non-cancer pain.

Mylan N.V. (MYL) has been seeking approval for its generic version of RELISTOR injection since 2015, and was given tentative approval by the FDA last May. Now that the Court has ruled in Progenics/ Valeant's favor, Mylan will have to wait until 2024 to launch generic RELISTOR.

Another trial is slated for June 4, 2018, and the court is expected to decide the infringement and validity of other patents that could prevent generic RELISTOR vials until 2029 and generic RELISTOR syringes until 2030.

Full-year 2017 net global sales of RELISTOR, as reported by Valeant, totaled $73.1 million compared to $70.6 million in 2016.

PGNX closed Wednesday's trading at $6.69, up 1.36%.

STAAR Surgical Co. (STAA) turned to profit in the first quarter of 2018 on 33% sales growth.

Net income for the first quarter of 2018 was approximately $0.6 million or $0.01 per share compared with a net loss of $2.2 million or $0.05 per share for the prior year quarter. Adjusted Net Income for the first quarter of 2018 was $1.8 million or $0.04 per share, compared to an Adjusted Net Loss in the prior year quarter of $1.6 million or $0.04 per share.

Net sales were $27.1 million for the first quarter of 2018, up 33% from $20.4 million reported in the prior year quarter.

STAA closed Wednesday's trading at $16.75, up 2.13%.