On May 2, 2019 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported a financial and pipeline update for the quarter ended March 31, 2019 (Press release, Acorda Therapeutics, MAY 2, 2019, View Source [SID1234535541]).

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"Since INBRIJA became commercially available at the end of February, the feedback from both healthcare professionals and people with Parkinson’s, or PWPs, has been enthusiastic, and our Prescription Support Services center has received approximately 2,000 prescription requests. Our market research has consistently shown that both physicians and PWPs consider OFF periods one of the most troubling and disruptive aspects of Parkinson’s, and that they regard INBRIJA as having the potential to address this significant unmet need," said Ron Cohen, M.D., Acorda’s President and CEO. "We are currently meeting with insurers to discuss formulary placement. We are also continuing to roll out in-person and digital programs to educate healthcare professionals and PWPs about INBRIJA."

First Quarter 2019 Financial Results

For the quarter ended March 31, 2019, the Company reported INBRIJA net revenue of $1.3 million. INBRIJA became commercially available on February 28, 2019.

For the quarter ended March 31, 2019, the Company reported AMPYRA net revenue of $40.1 million compared to $102.8 million for the same quarter in 2018. In September 2018, AMPYRA lost its exclusivity and generics entered the market. Consequently, the Company expects AMPYRA revenue to continue to decline.

Research and development (R&D) expenses for the quarter ended March 31, 2019 were $16.0 million, including $0.7 million of share-based compensation compared to $30.6 million, including $1.7 million of share-based compensation for the same quarter in 2018.

Sales, general and administrative (SG&A) expenses for the quarter ended March 31, 2019 were $52.7 million, including $2.8 million of share-based compensation compared to $47.6 million, including $4.2 million of share-based compensation for the same quarter in 2018.

Benefit from income taxes for the quarter ended March 31, 2019 was $0.7 million compared to a provision for income taxes of $3.5 million for the same quarter in 2018.

The Company reported a GAAP net loss of $47.6 million for the quarter ended March 31, 2019, or $1.00 per diluted share. GAAP net loss in the same quarter of 2018 was $8.2 million, or $0.18 per diluted share.

Non-GAAP net loss for the quarter ended March 31, 2019 was $26.5 million, or $0.56 per diluted share. Non-GAAP net income in the same quarter of 2018 was $6.8 million, or $0.14 per diluted share. This quarterly non-GAAP net (loss) income measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, and changes in the fair value of acquired contingent consideration. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At March 31, 2019, the Company had cash, cash equivalents and short-term investments of $343.3 million compared to $445.6 million at year end 2018. The decline in cash was due in large part to non-recurring payments of approximately $45 million that were made in the first quarter, primarily related to AMPYRA inventory purchases. The Company does not expect to purchase additional AMPYRA inventory in 2019.

For the remainder of 2019, the Company expects cash expenditures to align with normal operating activities, and continues to believe that it can become cash flow positive without raising additional capital, based on its long-term projections.

2019 Financial Guidance

During INBRIJA’s launch year, the Company does not expect to provide INBRIJA revenue guidance.

The Company will no longer provide revenue guidance for AMPYRA, due to the unpredictable trajectory of revenue decline given the entrance of generics.

R&D expenses for the full year 2019 are expected to be $70-$80 million and SG&A expenses for the full year 2019 are expected to be $200-$210 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."

First Quarter 2019 Highlights

INBRIJA (levodopa inhalation powder)

In March, the Company submitted responses to the INBRIJA Marketing Authorization Application (MAA) Day 120 list of questions. A final decision from the European Commission is expected before the end of 2019. Acorda is seeking approval to market INBRIJA in the European Union.

In March, new one-year safety and exploratory efficacy outcomes data from the extension of the Phase 3 SPANSM-PD trial were presented at the Academy of Managed Care Pharmacy (AMCP) Managed Care & Specialty Pharmacy Annual Meeting.

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 (866) 393-4306 (domestic) or (734) 385-2616 (international) and reference the access code 4783943. 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, 2019 until 11:59 p.m. ET on June 2, 2019. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international); reference code 4783943. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

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 loss, adjusted to exclude the items below, and has provided 2019 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 loss, 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, and acquired Biotie debt, and (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods. The Company believes its non-GAAP net loss 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 loss, we have provided 2019 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, 2019 Selecta Biosciences, Inc. (Nasdaq: SELB) ("Selecta"), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform technology, ImmTOR (SVP Rapamycin), reported that it plans to issue its first quarter 2019 financial results before the open of the U.S. financial markets on Thursday, May 09, 2019 (Press release, Selecta Biosciences, MAY 2, 2019, View Source [SID1234535574]).

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At 8:30 a.m. ET that day, Selecta will host a conference call and live audio webcast to discuss first quarter 2019 financial results and provide a corporate update. Investors and the public can access a live and archived webcast of this call via the Investors & Media section of the company’s website, View Source Individuals may also participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10127202.

On May 2, 2019 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, reported that the Company will release its first quarter 2019 financial results after the market closes on Thursday, May 9, 2019 (Press release, Synlogic, MAY 2, 2019, View Source [SID1234535589]). The press release will be followed by a conference call at 5:00 pm ET, which will be open to the public via telephone and webcast. During the conference call, the Company will review its financial results and provide a corporate update.

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The conference call dial-in numbers are (844) 815-2882 for domestic callers and (213) 660-0926 for international callers. The conference ID number for the call is 2154739. Participants may access the live webcast via a link on the Synlogic website in the Events Calendar of the Investors and Media section. For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Company’s website.

On May 2, 2019 Idera Pharmaceuticals, Inc. ("Idera") (NASDAQ: IDRA), a clinical-stage biopharmaceutical company focused on the development, and ultimately the commercialization, of therapeutic drug candidates for both oncology and rare disease indications, reported its financial and operational results for the first quarter ended March 31, 2019 (Press release, Idera Pharmaceuticals, MAY 2, 2019, View Source [SID1234535694]).

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"The first quarter of this year represented another period of solid execution from everyone on our team," stated Vincent Milano, Idera’s Chief Executive Officer. "During this quarter, we completed enrollment of ILLUMINATE-204, positioning ourselves to provide a full analysis from this trial in the fourth quarter of this year. We’ve also made significant progress with the enrollment of Idera’s first pivotal trial, ILLUMINATE-301, for which we expect to complete accrual during the fourth quarter. And, we recently presented the data from the ILLUMINATE-101 monotherapy trial at AACR (Free AACR Whitepaper), strengthening the body of translational and clinical evidence that tilsotolimod stimulates an immune response within the tumor microenvironment. We believe that this may be a key to enhancing the outcomes of existing immunotherapy regimens."

Milano continued, "Lastly, we’ve made great strides in preparing to initiate the expansion of activities for tilsotolimod through the ILLUMINATE-206 multi-cohort trial, which we anticipate will launch this quarter. We appreciate the financial commitment and teamwork from our collaboration partner, Bristol Myers Squibb, which assisted us in taking on this endeavor. Overall, this has been a highly productive quarter for our team and the momentum continues to swell as we advance towards these critical catalysts for our company."

ILLUMINATE (tilsotolimod) Clinical Development

ILLUMINATE 301 — Randomized phase 3 trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with PD-1 refractory metastatic melanoma:

· Overall Response Rate (ORR) and Overall Survival (OS) as primary endpoints;

· Trial initiated in the first quarter of 2018;

·Sites planned in 12 countries: 85 sites activated;

· Planned enrollment of approximately 300 patients; and

· Completion of enrollment expected during the fourth quarter of 2019.

ILLUMINATE 206 — Phase 2, multi-center trial to test the safety and effectiveness of tilsotolimod in combination with ipilimumab and nivolumab in treating patients with Squamous Cell Carcinoma of the Head and Neck (SCCHN) and Microsatellite Stable Colorectal Cancer (MSS-CRC).

· On March 11, 2019 entered into a second clinical trial collaboration with Bristol Myers Squibb (BMS) in which BMS has agreed to manufacture and supply YERVOY (ipilimumab) and OPDIVO (nivolumab) at its cost and for no charge for use in ILLUMINATE-206;

·Received notice from the U.S. Food and Drug Administration that we can proceed to implement the ILLUMINATE-206 clinical trial under a new Investigational New Drug (IND) application; and

· Both trial cohorts of SCCHN and MSS-CRC expected to initiate in the second quarter of 2019.

ILLUMINATE 204 — Phase 1/2 trial of tilsotolimod in combination with ipilimumab or pembrolizumab in patients with PD-1 refractory metastatic melanoma:

· Completed enrollment with 52 patients dosed in Phase 2 expansion at tilsotolimod 8 mg with ipilimumab;

·Completed target enrollment of at least 40 patients in the primary enrollment population constituting patients who are naïve to prior ipilimumab treatment in the metastatic setting;

· Presented an interim data update in December 2018 which showed:

· 32.4% ORR of the first 34 patients evaluable for efficacy including 9% (n=3) achieve Complete Response (CR); 24% (n=8) achieving Partial Response (PR); and 76.5% (n=26) achieving disease control (CR, PR or Stable Disease [SD]); and

· Final overall response rate (ORR) data from the ipilimumab combination arm of ILLUMINATE-204 expected in the fourth quarter of 2019.

ILLUMINATE 101 — Phase 1b trial of tilsotolimod monotherapy in patients with refractory solid tumors:

· Completed enrollment in all dose cohorts of the trial; and

· Poster presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2019 Annual Meeting on April 2, 2019 which demonstrated:

Safety Data

· No dose limiting toxicities or treatment-related adverse events were observed;

· No treatment-emergent adverse events (TEAEs) leading to treatment or study discontinuation or death occurred; and

· The most common grade 3/4 TEAEs were anemia, hyponatremia, pain, sepsis (n=3 each), fatigue and thrombocytopenia (n=2 each).

Efficacy Data

· Of 29 evaluable patients, 13 (45%) had a RECIST v1.1 disease assessment of stable disease (SD), with a disease control rate of 45%;

· Of the 13 patients with SD, 5 (38%) had maximum tumor shrinkage >10% below baseline;

· Duration of SD ranged from 1.3 to 9.7+ months from start of treatment, with 3 patients ongoing; and

· No correlations between dose and efficacy were observed.

Translational Data

· Fresh flow cytometry in 2 of 3 analyzed patients showed HLA-DR (MHC Class II) upregulation at 24 hours compared with pre-treatment; and

·Robust activation and upregulation of type I IFN pathway was observed across analyzed tumor types, demonstrated by increased IRF7, IFIT1, and IFIT2 gene expression, and early increases in type I IFN signaling.

Financial Results

First Quarter Results

Net loss applicable to common stockholders for the three months ended March 31, 2019 was $11.0 million, or $0.40 per basic and diluted share, compared to net loss applicable to common stockholders of $20.1 million, or $0.81 per basic and diluted share, for the same period in 2018. Revenue for each of the three months ended March 31, 2019 and 2018 was nominal. Research and development expenses for the three months ended March 31, 2019 totaled $8.1 million compared to $13.6 million for the same period in 2018. General and administrative expense for the three months ended March 31, 2019 totaled $3.1 million compared to $3.5 million for the same period in 2018. Merger-related costs, net for the three months ended March 31, 2018 totaled $3.4 million and related to our contemplated merger transaction. No such costs were incurred for the same period in 2019. Restructuring costs for the three months ended March 31, 2019 was nominal and related to our decision in July 2018 to wind-down our discovery operations. No such costs were incurred for the same period in 2018.

As of March 31, 2019, our cash, cash equivalents and investments totaled $59.9 million compared to $71.4 million as of December 31, 2018. We currently anticipate that, based on our current operating plan, our existing cash, cash equivalents and investments will fund our operations into the second quarter of 2020.

On May 2, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, May 9, 2019 to report its first quarter 2019 financial results and provide a corporate update (Press release, Blueprint Medicines, MAY 2, 2019, View Source [SID1234535575]).

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To access the live conference call, please dial (855) 728-4793 (domestic) or (503) 343-6666 (international), and refer to conference ID 9671728. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.