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Jazz Pharmaceuticals Announces FDA Acceptance of NDA for Solriamfetol (JZP-110) for Excessive Sleepiness Associated with Narcolepsy or Obstructive Sleep Apnea

On March 2, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the U.S. Food and Drug Administration (FDA) has accepted for filing with standard review the company’s New Drug Application (NDA) seeking marketing approval for solriamfetol, an investigational medicine for the treatment of excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea (OSA) (Press release, Jazz Pharmaceuticals, MAR 2, 2018, View Source;p=RssLanding&cat=news&id=2335912 [SID1234524340]). The Prescription Drug User Fee Act (PDUFA) goal date for an FDA decision is December 20, 2018.

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"We believe this medicine will provide a meaningful option for patients living with excessive sleepiness due to narcolepsy or OSA, and we look forward to working with the FDA during the review process for solriamfetol," said Karen Smith, M.D., Ph.D., executive vice president, research and development and chief medical officer at Jazz Pharmaceuticals. "Jazz continues to invest in ongoing research, education and advocacy on behalf of the sleep community, including studying solriamfetol for the treatment of excessive sleepiness in other areas of unmet need, such as Parkinson’s disease."

The solriamfetol Phase 3 clinical program includes one study evaluating excessive sleepiness in adult patients with narcolepsy (TONES 2), two studies evaluating excessive sleepiness in adult patients with OSA (TONES 3 and TONES 4), and an open-label, long-term safety and maintenance of efficacy study (TONES 5) in the treatment of excessive sleepiness in patients with narcolepsy or OSA.

About OSA and Excessive Sleepiness
OSA is a prevalent disease (as high as 14% in men and 5% in women) with excessive sleepiness being a major presenting complaint in many cases.1-2 Excessive sleepiness in OSA is associated with impairments in cognitive function, safety, productivity, interpersonal relationships, and overall quality of life. Positive Airway Pressure (PAP) therapy, with its most common form being Continuous Positive Airway Pressure (CPAP), has been shown to be an effective therapy for sleep-related airway obstruction, with frequent improvement in excessive sleepiness in many patients; however, not all patients tolerate CPAP therapy and among those who tolerate CPAP, usage is highly variable. It is estimated that excessive sleepiness persists in 13%–65% of people utilizing CPAP for OSA.3-5

About Narcolepsy
Narcolepsy is a debilitating neurological disorder characterized by excessive sleepiness, and the inability to regulate sleep-wake cycles normally.6 It affects an estimated one in 2,000 people in the United States, with symptoms typically appearing in early adulthood. It is estimated that more than 50% of patients with narcolepsy have not been diagnosed.7 Studies have shown it may take 10 years or more for people with narcolepsy to receive a correct diagnosis.8 Excessive sleepiness is the primary symptom of narcolepsy and is present in all people with the disorder.2 Excessive sleepiness is characterized by the inability to stay awake and alert during the day resulting in unplanned lapses into sleep or drowsiness.2,7,9

About Solriamfetol (JZP-110)
Solriamfetol (JZP-110) is a selective dopamine and norepinephrine reuptake inhibitor (DNRI) in development for treatment of excessive sleepiness in adult patients with narcolepsy, OSA, and Parkinson’s disease. In 2014, Jazz Pharmaceuticals acquired a license to develop and commercialize solriamfetol from Aerial Biopharma. Jazz Pharmaceuticals has worldwide development, manufacturing, and commercialization rights to solriamfetol, excluding certain jurisdictions in Asia. SK Biopharmaceuticals, the discoverer of the compound (also known as SKL-N05), maintains rights in 12 Asian markets, including Korea, China and Japan. Solriamfetol has orphan drug designation in the United States for narcolepsy.

TG Therapeutics, Inc. to Present at Upcoming Investor Conferences

On March 2, 2018 TG Therapeutics, Inc. (NASDAQ:TGTX) reported that Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, will be presenting at two upcoming investor conferences (Press release, TG Therapeutics, MAR 2, 2018, View Source [SID1234524343]). Presentation details are as follows:

The Raymond James & Associates’ 39th Annual Institutional Investors Conference, being held at the JW Marriott Orlando Grande Lakes in Orlando, Florida. The presentation is scheduled to take place on Tuesday March 6, 2018 at 2:50pm ET.
The Cowen 38th Annual Health Care Conference, being held at the Boston Marriott Copley Place in Boston, Massachusetts. The presentation is scheduled to take place on Monday March 12, 2018 at 1:30pm ET.
A live webcast of each presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com.

Reata Pharmaceuticals, Inc. Announces Fourth Quarter and Full Year 2017 Financial and Operating Results

On March 2, 2018 Reata Pharmaceuticals, Inc. (Nasdaq:RETA) (Reata or Company), a clinical-stage biopharmaceutical company, reported financial results for the fourth quarter and full year ended December 31, 2017, and provided an update on the Company’s business and product development programs (Press release, Reata Pharmaceuticals, MAR 2, 2018, View Source;p=RssLanding&cat=news&id=2335885 [SID1234524342]).

"In 2017, Reata made significant strides towards our goal of building a deep pipeline of late-stage therapeutics for rare and life-threatening diseases," said Warren Huff, Chief Executive Officer. "We entered 2017 with one pivotal trial in pulmonary arterial hypertension associated with connective tissue disease and a broad portfolio of exploratory Phase 2 studies from which we produced meaningful clinical data and launched pivotal trials in two additional rare diseases, Alport syndrome and Friedreich’s ataxia. We begin 2018 with these three pivotal programs in the clinic and a highly focused Phase 2 program in four rare forms of CKD underway."

Pipeline Highlights

In 2017, we launched and completed the Phase 2 portion of the Phase 2/3 CARDINAL study for bardoxolone methyl in patients with CKD caused by Alport syndrome. In the Phase 2 clinical trial, bardoxolone methyl demonstrated a statistically significant, mean increase from baseline in kidney function, as assessed by eGFR, at the 12 week endpoint. On the basis of the Phase 2 results, we initiated the Phase 3 portion of the CARDINAL trial, which will enroll approximately 150 patients with Alport syndrome. The United States Food and Drug Administration (FDA) has provided guidance that one year data from the ongoing Phase 3 portion of the trial demonstrating an improvement in retained eGFR, which is the increase in eGFR versus placebo after the patients have been taken off drug for four weeks, may support accelerated approval for bardoxolone methyl.

We began the Phase 2 PHOENIX study in patients with autosomal dominant polycystic kidney disease, IgA nephropathy, type 1 diabetic CKD, and focal segmental glomerulosclerosis. Each cohort will enroll approximately 25 patients to evaluate the safety and efficacy of bardoxolone methyl treatment for each rare form of CKD. Enrollment has begun in the trial for each of the four rare forms of CKD.

We reported positive proof-of-concept data in the MOXIe trial of omaveloxolone in Friedreich’s ataxia, and we began the registrational portion of MOXIe in 2017. Omaveloxolone demonstrated a statistically significant improvement in modified Friedreich’s Ataxia Rating Scale (mFARS) scores of 3.8 points (p=0.0001) at the optimal dose level versus baseline, and a placebo-corrected improvement in mFARS scores of 2.3 points (p=0.06) in Part 1 of the MOXIe trial. The FDA has confirmed that mFARS is acceptable as the primary endpoint for part 2 of MOXIe and that it may consider either accelerated or full approval based upon the overall results of the trial and strength of the data.

Anticipated Clinical Milestones in 2018 and 2019

One year retained eGFR benefit data for CARDINAL Phase 2 patients in the third quarter of 2018
12 week eGFR data from one or more cohorts of PHOENIX in the second half of 2018
CATALYST Phase 3 data in the second half of 2018, pending a sample size re-calculation in the second quarter of 2018 that could change expected timing
CARDINAL Phase 3 data in the second half of 2019
Data from the registrational part 2 of MOXIe in the second half of 2019
Fourth Quarter Results

The Company incurred operating expenses of $26.5 million for the quarter ended December 31, 2017, with research and development accounting for $20.4 million. This compares to operating expenses of $16.7 million for the same period of the year prior, when research and development accounted for $11.8 million. A net loss of $16.7 million was reported by the Company for the quarter ended December 31, 2017, equating to a loss of $0.64 per share, compared to net loss of $4.1 million or $0.19 per share in the same period of the year prior.

2017 Financial Results

As of December 31, 2017, the Company had $129.8 million in cash and cash equivalents. We believe our existing cash and cash equivalents, in combination with available debt and an expected milestone from Kyowa Hakko Kirin, will be sufficient to enable us to fund our operating expenses and capital expenditure requirements, assuming the CATALYST sample size re-calculation does not result in a sample size at the high end of the range, through registrational data from CATALYST in 2018, and both CARDINAL and MOXIe in the second half of 2019.

The Company incurred operating expenses of $95.0 million for the 12 months ended December 31, 2017, with research and development accounting for $71.3 million. This compares to operating expenses of $56.7 million for the same period of the year prior, when research and development accounted for $39.5 million. The 67% increase in operating expenses was primarily due to an 81% research and development expense increase consisting of $23.9 million in expanded clinical and manufacturing activities, primarily for CARDINAL, CATALYST, MOXIe, the extension trial for CATALYST and LARIAT and PHOENIX as well as increased costs in other clinical and preclinical programs. A net loss of $47.7 million was reported by the Company for the 12 month period ended December 31, 2017, equating to a loss of $1.99 per share, compared to net loss of $6.2 million or $0.31 per share in the year prior. The increased net loss was primarily due to the increased operating expenses and a decrease in the amount of deferred revenue recognized in 2017.

Molecular Templates Closes $10 Million Debt Facility with Perceptive Advisors

On March 2, 2018 Molecular Templates, Inc., (Nasdaq:MTEM) a clinical stage biopharmaceutical company focused on the discovery and development of Engineered Toxin Bodies (ETBs), a new class of targeted biologic therapies that possess unique mechanisms of action in oncology, reported the closing of a $10 million debt facility with Perceptive Advisors (Press release, Molecular Templates, MAR 2, 2018, View Source [SID1234524341]). The proceeds from the debt facility will be used to repay an existing debt facility with Silicon Valley Bank and to support the Company’s build out of its manufacturing facility.

"Molecular Templates’ ETB platform is enabling development of new and differentiated products for the treatment of cancer. Perceptive is delighted to provide debt financing to support the build out of Molecular Template’s manufacturing facility and the advancement of the Company’s pipeline of ETB product candidates," said Sam Chawla of Perceptive Advisors.

"We appreciate the support from Perceptive. This financing provides Molecular Templates with capital to support the build out of our GMP manufacturing facility in Austin, Texas. Having our own GMP facility should shorten the time from lead development to IND and allow us to better support our own pipeline as well as existing and prospective partnerships," said Eric Poma Ph.D., Chief Executive and Chief Scientific Officer of Molecular Templates. "We are highly focused on advancing our pipeline, with updated clinical results for MT-3724 expected in 1H18 and new IND filings for other pipeline programs expected by year-end."

10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

Nektar Therapeutics has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission .