On May 2, 2018 Acorda Therapeutics, Inc. (Nasdaq: ACOR)reported update for the quarter ended March 31, 2018 (Press release, Acorda Therapeutics, MAY 2, 2018, View Source [SID1234525937]).

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"With FDA’s acceptance of Acorda’s NDA for INBRIJA and a PDUFA date of October 5, 2018, we are focused on preparations for the potential U.S. approval and launch," said Ron Cohen, M.D., Acorda’s President and CEO. "We are also looking forward to the opportunity to argue our case for our AMPYRA patents at the U.S. Court of Appeals on June 7."

First Quarter 2018 Financial Results

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the quarter ended March 31, 2018, the Company reported AMPYRA net revenue of $102.8 million compared to $112.0 million for the same quarter in 2017. The quarter over quarter reduction in net revenue was primarily related to a modest expansion in customer inventories in the fourth quarter 2017 which normalized by the end of the first quarter 2018.

Research and development (R&D) expenses for the quarter ended March 31, 2018 were $30.6 million, including $1.7 million of share-based compensation compared to $46.5 million, including $2.5 million of share-based compensation for the same quarter in 2017. Quarter-over-quarter reductions in R&D expenses are primarily the result of our corporate restructuring which occurred in 2017.

Sales, general and administrative (SG&A) expenses for the quarter ended March 31, 2018 were $47.6 million, including $4.2 million of share-based compensation compared to $52.0 million, including $5.3 million of share-based compensation for the same quarter in 2017. Quarter-over-quarter reductions in SG&A expenses are primarily the result of our corporate restructuring which occurred in 2017.

Provision for income taxes for the quarter ended March 31, 2018 was $3.5 million, including $0.5 million of cash taxes, compared to a benefit from income taxes of $0.9 million, including $1.9 million of cash taxes for the same quarter in 2017.

The Company reported a GAAP net loss of $8.2 million for the quarter ended March 31, 2018, or $0.18 per diluted share. GAAP net loss in the same quarter of 2017 was $18.9 million, or $0.41 per diluted share.

Non-GAAP net income for the quarter ended March 31, 2018 was $6.8 million, or $0.14 per diluted share. Non-GAAP net loss in the same quarter of 2017 was $3.6 million, or $0.08 per diluted share. This quarterly non-GAAP net income (loss) measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, changes in the fair value of acquired contingent consideration, and acquisition-related expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At March 31, 2018, the Company had cash, cash equivalents and short-term investments of $333.0 million.

Guidance for 2018

The Company reiterates AMPYRA 2018 net revenue guidance of $330-$350 million. The Company expects to maintain exclusivity of AMPYRA at least through July 30, 2018; this guidance is subject to change based on the appellate court’s decision.
R&D expenses for the full year 2018 are expected to be $100-$110 million and include manufacturing expenses associated with INBRIJA. This guidance is a non-GAAP projection that excludes share-based compensation, as more fully described below under "Non-GAAP Financial Measures."
SG&A expenses for the full year 2018 are expected to be $170-$180 million. This guidance is a non-GAAP projection that excludes share-based compensation, as more fully described below under "Non-GAAP Financial Measures."
The Company expects to end 2018 with a projected year-end cash balance in excess of $300 million.
First Quarter 2018 Pipeline and Corporate Updates

INBRIJA (levodopa inhalation powder)
In February, the FDA notified the Company that it had accepted for filing the New Drug Application (NDA) for INBRIJA. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target date of October 5, 2018 for issuing its decision on the NDA.
In March, the Company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for INBRIJA. Acorda is seeking approval to market INBRIJA in the European Union.
In April, the Company presented new INBRIJA data from four accepted abstracts during two oral platform presentations at the American Academy of Neurology Annual Meeting in Los Angeles. A safety assessment in early morning OFF symptoms in patients with Parkinson’s disease (Study 009) was presented by Dr. Stuart H. Isaacson and Dr. Robert H. Hauser; long-term pulmonary safety and efficacy of inhaled levodopa in Parkinson’s disease (Study 005) was presented by Charles Oh, MD, VP Clinical Development at Acorda.
AMPYRA Patent Appeal
Oral argument before the U.S. Court of Appeals for the Federal Circuit has been scheduled for June 7, 2018.
rHIgM22 Update
Data from the rHIgM22 Phase 1 study in 27 people with acute relapsing multiple sclerosis showed that a single dose of rHIgM22 was not associated with any safety signals. The study’s primary endpoint was safety and tolerability of a single dose following a relapse.
The study was not powered to show efficacy and exploratory efficacy measures showed no difference between the treatment groups.
Webcast and Conference Call

The Company will host a conference call today at 8:30 a.m. ET. To participate in the conference call, please dial (833) 236-2756 (domestic) or (647) 689-4181 (international) and reference the access code 2477088. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on May 2, 2018 until 11:59 p.m. ET on June 2, 2018. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 2477088.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain historical and forward-looking non-GAAP financial measures. In particular, Acorda has provided non-GAAP net income, adjusted to exclude the items below, and has provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes the presentation of non-GAAP net income, when viewed in conjunction with our GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because this measure excludes (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) non-cash interest charges related to the accounting for our outstanding convertible debt which are in excess of the actual interest expense owing on such convertible debt as well as non-cash interest related to the Fampyra monetization, non-cash interest charges related to our asset based loan which was terminated in 2017 and acquired Biotie debt, (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods, and (iv) acquisition related expenses and related foreign currency losses and gains that pertain to a non-recurring event. The Company believes its non-GAAP net income measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding projected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

In addition to non-GAAP net income, we have provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Due to the forward looking nature of this information, the amount of compensation charges and benefits needed to reconcile these measures to the most directly comparable GAAP financial measures is dependent on future changes in the market price of our common stock and is not available at this time. The Company believes that these non-GAAP measures, when viewed in conjunction with our GAAP results, provide investors with a more meaningful understanding of our ongoing and projected R&D and SG&A expenses. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

On May 2, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) to the company’s lead phospholipid drug conjugate, CLR 131, for the treatment of neuroblastoma (Press release, Cellectar Biosciences, MAY 2, 2018, View Source [SID1234525957]).

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"Neuroblastoma is a devastating cancer most often found in infants and young children. The grant of RPDD for CLR 131 in conjunction with the orphan drug designation we received in March highlight the critical need for new treatments in the fight against this disease," said John Friend, M.D., chief medical officer of Cellectar. "We look forward to working with the FDA to bring this potential therapy to pediatric patients and expect to begin a clinical study in neuroblastoma during the second half of 2018."

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children from birth to 18 years old, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. If CLR 131 is approved by the FDA for neuroblastoma, the rare pediatric disease designation may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future NDA or BLA submission which would reduce the FDA review time from twelve months to six months. Currently, these vouchers can also be transferred or sold to another entity. Over the last 16 months, five priority review vouchers were sold for between $110 million to $150 million each.

About Neuroblastoma

Neuroblastoma, a neoplasm of the sympathetic nervous system, is the most common extracranial solid tumor of childhood, accounting for approximately 7.8% of childhood cancers in the United States and is recognized by the FDA as an orphan disease. The incidence is about 10.54 cases per 1 million per year in children younger than 15 years and 90% are younger than 5 years at diagnosis. Approximately 50% of patients present with metastatic disease requiring systemic treatment. Although the prognosis is favorable in children under one year of age with an 86% to 95% 5-year survival, in children aged one to 14 years the 5-year survival ranges from 34% to 68%.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. In the second half of 2018 the company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma, as well as a Phase 1 study in combination with external beam radiation for head and neck cancer.

On May 2, 2018 Sandoz, a Novartis division, reported that the US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the Biologics Licensing Application (BLA) for its proposed biosimilar rituximab (Press release, Novartis, MAY 2, 2018, View Source [SID1234525974]).

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Sandoz stands behind the robust body of evidence included in the regulatory submission and is currently evaluating the content of the letter. While disappointed, Sandoz remains committed to further discussions with FDA in order to bring this important medicine to US patients as soon as possible.

There is a substantial unmet need for access to safe and effective therapies. Sandoz is committed to increasing patient access to high-quality, life-enhancing biosimilar medicines. As the pioneer and global leader in biosimilar medicines, Sandoz has five biosimilar medicines currently marketed in various countries worldwide, as well as a leading global pipeline.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," "evaluating," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labelling for biosimilar rituximab, or any of the other investigational or approved biosimilar products described in this press release, or regarding potential future revenues from biosimilar rituximab and such other investigational and approved biosimilar products. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that biosimilar rituximab or any of the other investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time. Neither can there be any guarantee that if approved, biosimilar rituximab or such other biosimilar products will be approved for any or all of the indications in the respective reference product’s label. Nor can there be any guarantee that biosimilar rituximab, the other marketed products in the Sandoz biosimilar portfolio, or the potential products in the Sandoz biosimilar pipeline will be commercially successful in the future. In particular, management’s expectations regarding biosimilar rituximab and such other biosimilar products could be affected by, among other things, regulatory actions or delays or government regulation generally; the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; competition in general, including potential approval of additional versions of biosimilar rituximab or such other biosimilar products; the particular prescribing preferences of physicians and patients; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; litigation outcomes, including intellectual property disputes or other legal efforts to prevent or limit Sandoz from selling its biosimilar products; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

On May 2, 2018 MannKind Corporation(Nasdaq:MNKD) reported that it will release its 2018 first quarter financial results on Wednesday, May 9, 2018 and its management will host a conference call to discuss the financial results and other Company developments at 5:00 PM (Eastern Time) on May 9, 2018 (Press release, Mannkind, MAY 2, 2018, View Source [SID1234525992]).

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Presenting from the Company will be its Chief Executive Officer, Michael Castagna, Chief Financial Officer, Steven Binder, Chief Commercial Officer, Pat McCauley and Chief Medical Officer, David Kendall.

To view and listen to the earnings call webcast live via the Internet, visit the Company’s website at www.mannkindcorp.com and click on the "Q1 2018 MannKind Earnings Conference Call" link in the Webcasts section of News & Events. To participate in the live call by telephone, please dial (800) 289-0438 toll-free or (323) 794-2423 toll/international and use the conference passcode: 3321662.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 toll-free or (412) 317-6671 toll/international and use the replay passcode: 3321662. A replay will also be available on MannKind’s website for 14 days.

On May 2, 2018 OnKure, Inc., the epigenetics–focused drug discovery and development company that is advancing the first Largazole-derived, histone deacetylase inhibitor into Phase 1 clinical trials, reported it has completed a $7M Series A financing (Press release, OnKure, MAY 2, 2018, View Source [SID1234535520]). Delian Capital led the investment round with participation from existing and new investors including OnKure management. The investment will help the company aggressively accelerate its research and development activities.

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Delian Capital invests in early stage biopharmaceutical startups that redefine or create large new markets, with notable investments such as Chance Pharma, MicuRx and Bioceutiq. The Series A financing builds on an exceptional year for OnKure, which made significant progress towards advancing its first clinical candidate, OKI-179, towards Phase 1.

OnKure’s platform transforms naturally occurring, biologically active lead compounds into commercial drugs that address a variety of diseases with an emphasis on oncology. At the heart of OnKure’s solution to Class 1 histone deacetylase inhibition is a molecule (OKI-179) that parallels the potency and selectivity of Romidepsin (Istodax), the most potent Class 1 biased histone deacetylase inhibitor currently approved by the FDA. While Romidepsin is administered by continuous infusion, OKI-179 was designed as an orally active drug and has demonstrated a superior safety profile in preclinical studies. As an orally administered therapy, it is also more suitable for combination dosing with a variety of targeted anti-cancer agents.

"We are delighted that Delian Capital chose to lead our Series A round and I look forward to working with Mr. Guobao Zhao, Vice President of Delian Capital’s Healthcare Division and OnKure’s newest addition to the board," said Tony Piscopio, co-founder, President & CEO of OnKure. "OnKure will use these funds to accelerate the clinical development of OKI-179 and expand its pipeline through in-licensing first–in–class or best–in–class compounds that synergize with OKI-179."

Mr. Guobao Zhao, Vice President of Delian Capital, commented, "Delian invests in pharmaceutical companies with a deep understanding of disease mechanisms and extensive clinical expertise." Mr. Zhao added, "OnKure has a very experienced management team, and thus we look forward to supporting the parallel development of OKI-179 in the United States and China, as this novel HDAC inhibitor has the potential to offer an attractive option for addressing cancer’s growing unmet medical needs."