On May 5, 2020 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported its financial results for the first quarter ended March 31, 2020 (Press release, Acorda Therapeutics, MAY 5, 2020, View Source [SID1234557017]).

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"As we continue navigating the unprecedented operating environment created by COVID-19, our entire team remains focused on ensuring that the Parkinson’s disease and multiple sclerosis communities have continued access to Acorda’s critical medications," said Ron Cohen, M.D., Acorda’s President and Chief Executive Officer. "I am proud of how rapidly our team has evolved our business practices and developed new ways of working that enable us to keep our Acorda associates safe, while continuing to manufacture therapies that improve the lives of patients."

Dr. Cohen continued, "Despite significant disruption in the healthcare sector during the first quarter, INBRIJA and AMPYRA sales were consistent with our expectations, which took into account typical seasonal variability. We remain confident in our full-year expectations for AMPYRA, which is an established franchise supported by ongoing prescription renewals. For INBRIJA, which is a newer product driven by growth in new prescriptions, we are withdrawing our 2020 guidance. Due to COVID-19 stay-at-home orders and a widespread decrease in physician office visits, we do not have visibility into how changes in patient behavior may impact new prescription starts over the remainder of the year."

First Quarter 2020 Business Update

In response to the COVID-19 pandemic, Acorda launched a new, remote communications program, enabling it to communicate effectively and safely with healthcare professionals and patients to provide product education, information and support. The Company also launched virtual speaker programs and product webinars with meaningful attendance, including an Inbrija patient program that had over 500 participants. In addition, Acorda launched a new digital marketing program to drive awareness and educate people with Parkinson’s about INBRIJA.

First Quarter 2020 Financial Results

For the quarter ended March 31, 2020, the Company reported INBRIJA net revenue of $4.4 million, compared to $1.3 million for the same quarter in 2019.

For the quarter ended March 31, 2020, the Company reported AMPYRA net revenue of $20.1 million compared to $40.1 million for the same quarter in 2019. 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, 2020 were $7.7 million, including $0.4 million of share-based compensation compared to $16.0 million, including $0.7 million of share-based compensation for the same quarter in 2019.

Sales, general and administrative (SG&A) expenses for the quarter ended March 31, 2020 were $41.1 million, including $1.5 million of share-based compensation compared to $52.7 million, including $2.8 million of share-based compensation for the same quarter in 2019.

Change in fair value of derivative liability for the quarter ended March 31, 2020 was $26.5 million compared to $0 for the same quarter in 2019.

Benefit from income taxes for the quarter ended March 31, 2020 was $7.0 million compared to a benefit from income taxes of $0.7 million for the same quarter in 2019.

The Company reported a GAAP net loss of $6.5 million for the quarter ended March 31, 2020, or $0.14 per diluted share. GAAP net loss in the same quarter of 2019 was $47.6 million, or $1.00 per diluted share.

Non-GAAP net loss for the quarter ended March 31, 2020 was $24.4 million, or $0.51 per diluted share. Non-GAAP net loss in the same quarter of 2019 was $26.5 million, or $0.56 per diluted share. This quarterly non-GAAP net 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, asset impairment charges, changes in the fair value of the derivative liability, and expenses that pertain to a non-routine restructuring event. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At March 31, 2020, the Company had cash, cash equivalents, short-term investments and restricted cash of $126.3 million compared to $168.9 million at year end 2019. Restricted cash includes $42.7 million in escrow related to the 6% semi-annual interest portion, payable in cash or stock, of the convertible note exchange completed in December 2019. If the Company elects to pay interest due in stock, the restricted cash will be released from escrow.

2020 Financial Guidance

For the full-year 2020, Acorda continues to expect AMPYRA net revenue to be $85 – $110 million, and operating expenses to be $170 – $180 million. The operating expense guidance is a non-GAAP projection that excludes restructuring costs and share-based compensation as more fully described below under "Non-GAAP Financial Measures."

As a result of declines in physician office visits due to COVID-19 stay-at-home orders, the Company is withdrawing its previously announced 2020 INBRIJA net revenue guidance. This also necessitates the withdrawal of 2020 total net product revenue. Acorda continues to expect INBRIJA peak sales to be $300 – $500 million.

Webcast and Conference Call

The Company will host a conference call today at 4:30 p.m. ET. To participate in the conference call, please dial (833) 236-2756 (domestic) or (647) 689-4181 (international) and reference the access code 7999873. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 8:30 p.m. ET on May 5, 2020 until 11:59 p.m. ET on June 5, 2020. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 7999873. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On May 5, 2020 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST), a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported that the Company will participate in two upcoming virtual investor conferences. Jigar Raythatha, CEO, will present at (Press release, Constellation Pharmaceuticals, MAY 5, 2020, View Source [SID1234557016]):

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The Bank of America Merrill Lynch Global Health Care Conference at 9:00 AM EDT on May 12
The RBC Capital Markets Global Healthcare Virtual Conference at 10:55 AM EDT on May 20
Live audio webcasts of Mr. Raythatha’s presentation and archives for replay will be available on the Investor Relations section of Constellation’s website at View Source The audio webcast replay will be available for 90 days following the live presentation.

On May 5, 2020 New research from Exact Sciences Corp. (NASDAQ: EXAS) reported that uses real-world-adjusted adherence rates in a colorectal cancer microsimulation, the Colorectal Cancer and Adenoma Incidence and Mortality model (CRC-AIM), to reinforce the value of Cologuard (mt-sDNA) as an effective colorectal cancer screening test (Press release, Exact Sciences, MAY 5, 2020, View Source [SID1234557015]). Cologuard offers significant advantages over the fecal immunochemical test (FIT) and real-world adherence data further highlights those advantages. Cologuard has higher sensitivity, a longer interval, and higher observed adherence rates in real-world usage. The modeling also highlights the importance of improving access to colonoscopy following a positive Cologuard or FIT test.

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These results were accepted for presentation at DDW 2020 in a series of abstracts. They are now available on the DDW website.

Colorectal cancer (CRC) is the second leading cause of cancer death for men and women, in part because many cancers go undetected until later stages when treatment options are limited.1 Colorectal cancer can be prevented or detected early through screening; however, approximately 45 million Americans are not up to date with CRC screening.2

"Policymakers and other leaders often rely on modeling to make decisions about colorectal cancer screening," said Dr. Paul Limburg, Chief Medical Officer of Exact Sciences’ screening business. "Modeling that incorporates real-life patient behaviors, and the impact of those behaviors on health outcomes, is limited. This new suite of abstracts provides insight into the effect that complex and dynamic patient patterns have on colorectal cancer screening outcomes."

Included below are titles, abstract numbers, and a brief summary of each abstract accepted for presentation at DDW.

Estimating the Impact of Imperfect Adherence to Stool-based Colorectal Cancer Screening Strategies on Comparative Effectiveness using the CRC-AIM Microsimulation Model
Abstract Number: Mo1598

Real-life adherence rates for CRC screening are imperfect and can vary widely by modality, health system setting, and use of patient compliance navigation programs. Cologuard (mt-sDNA) was recently shown to have high population-level adherence in a Medicare population, which researchers theorized was attributable to its noninvasive approach, widespread accessibility, and embedded patient navigation system. Using real-world adherence rates of 70% for mt-sDNA3 and 40-50% for FIT,4 the CRC-AIM microsimulation model estimated that the use of mt-sDNA resulted in an 8.4% to 19.1% increase in life-years gained (LYG) compared with FIT for screening ages 50-75 years.

More Fecal Immunochemical Tests are Needed to Match the Clinical Benefit of Equivalent Numbers of Multitarget Stool DNA Tests: CRC-AIM Microsimulation Model Results
Abstract Number: Su1773

Patient adherence to the annual testing regimes of FIT has proven difficult to achieve in real world settings.5 Predicted outcomes of annual FIT and triennial mt-sDNA were simulated for 4 million individuals between 50 and 75. At least twice as many FIT tests are needed to match the same clinical benefit compared with mt-sDNA testing (21 vs. 9) using guideline recommended CRC screening initiation age and test interval.

Microsimulation Study of Life-Years Gained from Screening vs. Follow-up Colonoscopy using the CRC-AIM Model
Abstract Number: Sa1658

Health care providers often face a challenge ensuring patient adherence to follow-up colonoscopy after a positive stool-based test. Current reimbursement and coverage policies can trigger patient cost-sharing when patients seek necessary follow-up colonoscopy or when a screening colonoscopy turns diagnostic, depending upon health insurance benefits. In a third study leveraging the CRC-AIM model, the analysis showed that there are approximately 3-to-5 times more LYG per colonoscopy when the colonoscopy was performed as a diagnostic follow-up to a positive stool-based test rather than as a screening colonoscopy alone.

"All too often, colonoscopies following a positive stool-based test and screening colonoscopies that turn diagnostic do not receive full reimbursement from insurers, which can prevent patients from scheduling them," said Kevin Conroy, chairman and CEO. "These data show the urgent need to remove cost barriers for these colonoscopies as they have a measurable positive impact on patient lives."

Higher Impact on Clinical Outcomes from Delays in Colorectal Cancer Screening with the Fecal Immunochemical Test vs. Multitarget Stool DNA: CRC-AIM Microsimulation Model Results
Abstract Number: Tu1822

One obstacle to CRC screening is a delay in the start of screening or a lack of adherence to repeat testing. For example, screening rates among people 50 to 54 years old are only 50%, according to recent CDC data.6 The assumption in this modeling analysis was that recommended triennial mt-sDNA or annual FIT was delayed by 12, 18, or 24 months every time screening was due. The reduction in CRC-related mortality with a 12, 18, and 24-month delay for patients 50 to 75 was greater with triennial mt-sDNA (67%, 63%, and 59%, respectively) than annual FIT (65%, 59%, and 53%). The relative efficacy of mt-sDNA was better than FIT when considering these clinically realistic delayed screening adherence scenarios.

To promote transparency and the credibility of this new model, Exact Sciences has made available CRC-AIM’s formulas and parameters at View Source so other researchers and members of the modeling community can address outstanding questions related to CRC screening.

On May 5, 2020 Portage Biotech Inc. ("Portage" or the "Company") reported an update on a subsidiary company, Saugatuck Therapeutics Ltd. ("Saugatuck") (Press release, Portage Biotech, MAY 5, 2020, View Source [SID1234557014]). Portage Biotech Inc. holds an 70% equity interest in Saugatuck.

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Portage is pleased to announce that initial proof of concept of the nanolipogel ("NLG") formulation has been achieved with the initial investment. This has triggered the next tranche of capital infusion of $700,000 USD. Saugatuck has been able to formulate a proprietary PD1 aptamer in the NLG formulation and have shown the formulation properly modulates PD1 signaling. In non clinical in vivo experiments, the NLG-PD1 performed favorably compared to a mouse PD1 antibody. The additional founding will support exploration of multiple PD1 based co-formulations with small molecules and other DNA aptamers.

Separately, this work has triggered a license from D5 pharma to create additional proprietary DNA aptamers for immune-oncology targets. This license sits in another Portage company, Oncomer. Oncomer supplies Saugatuck with aptamers to be formulated in the NLG platform.

Dr. Ian Walters, CEO of Saugatuck and Portage commented" Most cancers are treated with multiple agents. Our co-formulation platform leverages the ability to modulate several pathways in a single product and direct its distribution to tumors. I am excited to begin testing our next wave of combinations in animal models and prioritizing our first clinical candidate."

On May 5, 2020 NETRIS Pharma SAS, a private clinical-stage biopharmaceutical company developing therapeutics based on dependence receptor biology, reported that it has entered into a clinical collaboration agreement with MSD to investigate the safety, clinical and biological activity of NP137 with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in patients with locally advanced/metastatic uterine tumors (Press release, Netris Pharma, MAY 5, 2020, View Source [SID1234557013]). NP137 targets netrin-1, which is overexpressed in over two-thirds of uterine tumors.

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The companies have entered this collaboration based on promising clinical data obtained from a phase 1 trial investigating NP137 monotherapy in patients with advanced solid tumors and the growing evidence that resistance to an anti-PD-1 therapy, such as KEYTRUDA, can be alleviated when combined with blocking netrin-1.

Under the terms of the agreement, NETRIS will sponsor a large phase 1b/2 study, which will be conducted in collaboration with the Centre Léon Bérard and with support of the specialist French oncology group of clinicians, GINECO (Groupe d’Investigateurs National des Etudes des Cancers Ovariens). The study will investigate the safety and efficacy of NP137 combined with KEYTRUDA in patients with advanced/metastatic endometrial carcinoma or cervix carcinoma.

"We are excited to collaborate with MSD, an established leader in cancer immunotherapy, on our proof-of-concept phase1b/2 study as we work to improve the lives of cancer patients," said Patrick Mehlen, Founder and Chief Executive Officer of NETRIS Pharma. "Immunotherapies are revolutionizing the treatment of patients in several cancer indications, but there remain many other tumor types in which existing immunotherapies have not demonstrated sufficient efficacy. Based on our preclinical and clinical data demonstrating the role of netrin-1 in promoting tumor progression and modulating tumor plasticity and microenvironment, we believe NP137’s effect in combination with KEYTRUDA will lead superior patient response to immunotherapy."

"The preliminary safety data and anti-tumor activity observed in the Phase 1a trial, together with the unique mode of action of NP137, are very exciting and clearly support evaluating a combination with KEYTRUDA," said Isabelle Ray Coquard, MD, Ph.D. and Principal Investigator of the trial. "We anticipate starting the study summer 2020."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp, a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

The Phase 1b/2 clinical trial will enroll up to 240 patients and be divided into a Ph1b part to evaluate the safety, tolerability, and pharmacokinetics/pharmacodynamics of the NP137 KEYTRUDA combination and/or chemotherapeutic agents. The Phase 2 part will assess via randomization the clinical activity of the combination in both tumor types.

About NP137

NP137, a humanized monoclonal antibody of isotype IgG1 directed against netrin-1, is the first drug candidate developed by NETRIS Pharma. Most types of tumors produce an abnormal amount of dependence receptors’ ligands, which prevents cells from dying. Netrin-1 is overexpressed in a large percentage of human cancers, including over two thirds of gynecologic cancers.

In preclinical studies, NP137 inhibited tumor growth and had a significant impact on tumoral plasticity, which potentiates the efficacy of chemotherapies and immune checkpoint inhibitors. In the phase 1 dose-escalation study, NP137 was found to be safe and very well tolerated up to 20mg/kg, with no dose limiting toxicity (DLT). In addition, patients with advanced uterine cancers exhibited encouraging signs of anti-tumor activity, including prolonged stable disease and objective responses.

About GINECO

GINECO (Groupe d’Investigateurs National pour l’Etude des Cancers de l’Ovaire et du sein) is the French Cooperative Group in Oncology labelled by INCA (National Cancer Institute in France) developing and conducting gynecological and metastatic breast cancer clinical trials at the national and international level. The network comprises more than 700 specialized investigators representing more than 150 public or private oncology units.