On May 11, 2020 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended March 31, 2020 and provided a corporate update (Press release, DURECT, MAY 11, 2020, View Source [SID1234557507]).

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Total revenues were $2.8 million and net loss was $9.9 million for the three months ended March 31, 2020 as compared to total revenues of $4.1 million and net loss of $7.1 million for the three months ended March 31, 2019.

At March 31, 2020, cash and investments were $52.5 million, compared to cash and investments of $64.8 million at December 31, 2019. Debt at March 31, 2020 was $20.4 million, compared to $20.3 million at December 31, 2019.

"We are pleased to be working with the FDA on the design of a double-blind, multi-center, placebo-controlled clinical study to evaluate the safety and efficacy of DUR-928 in COVID-19 patients with acute liver or kidney injury," stated James E. Brown, D.V.M., President and CEO of DURECT. "The first quarter was largely about adjusting to the challenges posed by the COVID-19 pandemic and planning our next clinical study of DUR-928 in alcoholic hepatitis, while continuing to make progress on our other key programs. In addition to initiating discussions with the FDA regarding the potential use of DUR-928 in COVID-19 patients, we were fortunate during Q1 to be able to complete enrollment in our NASH trial and respond to several information requests from the FDA as they continue their review of our New Drug Application (NDA) for POSIMIR."

Update on Selected Programs:

Epigenetic Regulator Program. DUR-928, the lead product candidate in the Company’s Epigenetic Regulator Program, is an endogenous, orally bioavailable, first-in-class small molecule, which may have broad applicability in acute organ injuries such as alcoholic hepatitis (AH) and COVID-19 patients with acute liver or kidney injury as well as in chronic liver diseases such as non-alcoholic steatohepatitis (NASH).

Clinical Trials

COVID-19

We are working with the FDA on the design of a double-blind, placebo-controlled, multi-center, proof-of-concept Phase 2 study to evaluate the safety and efficacy of DUR-928 in approximately 80 COVID-19 patients with acute liver or kidney injury.

Coronavirus disease 2019 (COVID-19) is an infectious disease caused by severe acute respiratory syndrome coronavirus (SARS-COV-2). The rapid spread of the disease has resulted in a pandemic with more than 4 million confirmed cases and over 280,000 deaths worldwide, over 79,000 of which have occurred in the United States as of this writing. While most cases result in mild symptoms, including fever, cough and shortness of breath, some progress into severe pneumonia and multi-organ failure, potentially as a result of severe immune overreaction (a cytokine storm), or as a result of ischemic injury, or other complications. Several studies reported that up to half of hospitalized patients with COVID-19 had elevated liver enzyme levels that signal liver injury and more than a third of hospitalized patients had kidney damage.

The reasons for potentially testing DUR-928 in this patient population include:

DUR-928 has demonstrated, both in vitro and in vivo, its ability to stabilize mitochondria, modulate inflammatory responses, and promote cell survival and tissue regeneration, which may render it to be effective in preventing or treating acute organ injury.

Patients with severe COVID-19 can develop multi-organ injury, including acute kidney, liver and/or cardiac injury, in addition to lung injury and acute respiratory distress syndrome (ARDS). Therefore, one could potentially save lives of those hospitalized patients with COVID-19 if one could prevent or treat acute organ injury by alleviating acute cell injury, regulating inflammation, promoting cell survival, and stimulating tissue regeneration.

Acute liver or kidney injury is a risk factor for poor outcomes in COVID-19 patients. These patients are being excluded from many ongoing anti-viral COVID-19 trials and are in great need of a new therapy.

Most relevant to COVID-19 patients with acute liver or kidney injury are results from the recently completed Phase 2a study in AH patients (see below). All 19 patients dosed with DUR-928 survived the 28-day study, while the historical 28-day mortality rate in AH patients is 26% on average.

To date, DUR-928 has been dosed in more than 280 subjects, both healthy volunteers and patients, in multiple Phase 1 and 2 studies and has been well tolerated. No serious adverse events have been associated with the drug.

Alcoholic Hepatitis (AH)

During 2019, we completed a Phase 2a clinical trial of DUR-928 in patients with AH. All 19 patients treated with DUR-928 in the AH trial survived the 28-day follow-up period and there were no drug-related serious adverse events. The study results were presented as a late-breaking oral presentation at The Liver Meeting 2019 by Dr. Tarek Hassanein, one of the trial’s principal investigators. In a separate poster presentation, Dr. Craig McClain presented additional comparative data from the Phase 2a clinical trial of DUR-928 and a control group of severe AH patients treated with corticosteroids in a contemporaneous AH trial conducted at University of Louisville. Additionally, the DUR-928 results were selected for inclusion in the "Best of The Liver Meeting" summary slide deck in the Alcohol-related Liver Disease category. Inclusion in this slide deck is considered a singular honor and indicates the high level with which the AASLD review committee regarded this study.

AH is an acute form of alcoholic liver disease (ALD) associated with long-term heavy intake of alcohol, and often occurs after a recent period of increased alcohol consumption. AH is typically characterized by recent onset jaundice and hepatic failure. An analysis of 77 studies published between 1971 and 2016, which included data from a total of 8,184 patients, showed the overall mortality from AH was 26% at 28 days. According to the most recent data provided by the Agency for Healthcare Research and Quality (AHRQ), a part of the US Department of Health and Human Services (HHS), there were over 117,000 hospitalizations for patients with AH in 2016. From a recent publication analyzing the mortality and costs associated with AH, the cost per patient is estimated at over $50,000 in the first year. ALD is one of the leading causes of liver transplants in the U.S., costing over $800,000 per patient.

We are working with the FDA and our advisors to finalize the design of a multi-center, international, randomized, double blind, placebo-controlled Phase 2b clinical trial of DUR-928 in severe AH patients. Patients in the trial will be randomized to receive 30 mg of DUR-928, 90 mg of DUR-928 or placebo. The primary goal of the trial will be to demonstrate a superior survival rate for patients treated with DUR-928 compared to those treated with placebo. Further details of the trial design, including the size of the trial and details on the endpoints will be provided at a future date. Due to the COVID-19 pandemic, we are updating our guidance for initiation of this trial and now expect the trial to begin enrolling patients in the second half of 2020.

Non-Alcoholic Steatohepatitis (NASH)

We have completed enrollment in the ongoing NASH trial. 62 patients have completed dosing and their final visits. Clinical data from the last few patients are being collected, while only a few were unable to complete final visits due to COVID-19 related office closings and travel restrictions. The Company remains on track to announce top-line study results mid-year.

The trial is a Phase 1b randomized and open-label clinical study conducted in the U.S. to evaluate safety, pharmacokinetics and signals of biological activity (including clinical chemistry and biomarkers as well as liver fat content and liver stiffness by imaging) of DUR-928 in NASH patients with stage 1-3 fibrosis. DUR-928 (at doses of 50 mg QD, 150 mg QD or 300 mg BID) is administered orally for 28 consecutive days and patients are followed up for an additional 28 days.

Non-alcoholic fatty liver disease (NAFLD) is the most common form of chronic liver disease in both children and adults. It is estimated that NAFLD affects approximately 30% to 40% of adults and 10% of children in the United States. NASH, a more severe and progressive form of NAFLD, is one of the most common chronic liver diseases worldwide, with an estimated prevalence of 3-5% globally. No drug is currently approved for NAFLD or NASH.

POSIMIR (bupivacaine extended-release solution) Post-Operative Pain Relief Depot. POSIMIR is the Company’s investigational post-operative pain relief depot that uses the Company’s patented SABER technology and is designed to deliver bupivacaine to provide up to 3 days of pain relief after surgery.

Since the Anesthetic and Analgesic Drug Products Advisory Committee (AADPAC) meeting on January 16, 2020, we have continued to interact with the FDA as they continue their review of the POSIMIR NDA.

The efforts to evaluate the program, develop a strategy for filing the response to the Complete Response Letter (CRL), and preparing the response, have been under the direction of Dr. Lee Simon, who was formerly the FDA’s Division Director of Analgesic, Anti-inflammatory and Ophthalmologic Drug Products. Dr. Simon also led our preparation efforts for the Advisory Committee meeting.

POSIMIR has not been approved by the FDA for marketing in the U.S. or elsewhere for any indication and there can be no assurance that the FDA will approve the submission described above.

Conference Call

We will host a conference call today at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss first quarter 2020 results and provide a corporate update:

Monday May 11th @ 4:30pm Eastern Time / 1:30 p.m. Pacific Time

Toll Free:877-407-0784

International:201-689-8560

Conference ID:13703442

Webcast:View Source

A live audio webcast of the presentation will also be available by accessing DURECT’s homepage at www.durect.com and clicking "Investors." If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under "Event Calendar" in the "Investors" section.

On May 11, 2020 Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers, reported recent highlights and developments as well as financial results for the first quarter ended March 31, 2020, which include (Press release, Omeros, MAY 11, 2020, View Source [SID1234557506]):

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Revenues for the first quarter of 2020 were $23.5 million, compared to $21.8 million and $33.4 million in the first and fourth quarters of 2019, respectively. The decrease from the fourth quarter of 2019 reflects the COVID-19-related postponement of cataract procedures by ASCs and hospitals in early March.

Net loss in the first quarter of 2020 was $29.0 million, or $0.53 per share, including non-cash expenses of $6.4 million, or $0.12 per share. This compares to a net loss of $24.3 million, or $0.50 per share, which included non-cash expenses of $6.0 million, or $0.12 per share, for the comparable quarter in 2019.

At March 31, 2020, Omeros had cash, cash equivalents and short-term investments available for operations of $54.0 million, a decrease of $6.8 million from December 31, 2019.

Omeros submitted the second part of its rolling biologics license application (BLA) for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). The submission was on schedule and consisted of information relating to the chemistry, manufacturing and controls (CMC) of narsoplimab.

"I am immensely proud of how Omeros’ employees have responded to the unprecedented challenges presented by the global pandemic, adapting to a changing work environment while continuing to meet program milestones with the same commitment, sense of urgency and level of productivity," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "We already are seeing rapid resumption of OMIDRIA purchases by ASCs and hospitals as they reopen and begin addressing the backlog of cataract surgery patients. For narsoplimab, we just submitted the second part of our rolling BLA as scheduled and continue to target next quarter for its completion. Our ongoing and upcoming clinical programs have weathered COVID-19 well, and we continue to target the start of our OMS906 clinical program next month and data readout from our ARTEMIS-IGAN trial next year. Our research laboratories and nonclinical functions have also remained fully operational. Our preparations for the commercial launch of narsoplimab have accelerated and, with the addition of recent hires, we continue to build top-tier sales, marketing and medical affairs teams. We look forward to adding narsoplimab to what we expect will be a long line of commercial products."

First Quarter and Recent Developments

Recent developments regarding OMIDRIA include the following:

Previously reported peer-reviewed published data demonstrate that OMIDRIA significantly reduces the need for intraoperative fentanyl, a potent and highly addictive opioid, while significantly decreasing pain scores. A separate study to assess the effect of OMIDRIA on use of postoperative opioid use was also recently completed. In that study an analysis of claims data over a 3-year period was performed by IBM Watson Health. Claims data were evaluated from 218,672 patients 65 years of age or older who

underwent cataract surgery. All patients were required to have no opioid use during the 6 months prior to surgery. Filled opioid prescriptions in the OMIDRIA-treated group were compared to those in the non-OMIDRIA-treated group. Patients who received OMIDRIA during surgery received fewer opioid pills in the 2 days and 7 days post-surgery than patients who did not receive OMIDRIA. The median reductions seen were 56 percent (20 pills versus 45 pills) at 2 days (p = 0.015) and 33 percent (40 pills versus 60 pills) at 7 days (p = 0.029). These data provide further evidence that OMIDRIA not only reduces the need for intraoperative fentanyl but also decreases the use of postoperative opioids.

The results of a retrospective study of the incidence of postoperative clinical cystoid macular edema (CME), breakthrough iritis, pain and photophobia in patients receiving OMIDRIA were published in the peer-reviewed Journal of Cataract and Refractive Surgery. The study demonstrated with statistical significance that patients receiving OMIDRIA had lower incidences of clinical CME (p = 0.021), breakthrough iritis (p = 0.001) and pain (p = 0.001) compared to a control group receiving conventional perioperative steroids. Patients receiving OMIDRIA also had lower incidence of photophobia, though not statistically significant.

A manuscript on the safety and efficacy of OMIDRIA for pediatric cataract surgery was also published by Journal of Cataract and Refractive Surgery. The randomized double-blind study in children undergoing cataract surgery showed that OMIDRIA is safe to use in children and resulted in lower pain scores for patients receiving OMIDRIA than for the control group. As a result, the FDA-approved label for OMIDRIA has no age restriction

Recent developments regarding narsoplimab, Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) in Phase 3 clinical programs for the treatment of HSCT-TMA, Immunoglobulin A (IgA) nephropathy, and atypical hemolytic uremic syndrome (aHUS), include the following:

Omeros submitted the second part of its rolling BLA for narsoplimab for the treatment of HSCT-TMA, consisting of CMC-related information, including data on manufacturing, analytical procedures, and associated method validations.

A manuscript authored by a group from the University of Leicester led by Dr. Jonathan Barratt PhD, FCRP, Professor of Renal Medicine, has been accepted for peer-reviewed publication. The manuscript describes the beneficial effects of narsoplimab in IgA vasculitis-associated nephritis, a rapidly progressive glomerulonephritis. A second manuscript presenting Omeros’ IgA nephropathy Phase 2 clinical data and authored by the company’s IgA nephropathy Academic Leadership Committee, which is comprised of international thought leaders, has also undergone journal review and is expected to be published soon.

Consistent with FDA guidelines and recommendations of the independent data safety monitoring committee regarding ongoing clinical trials during the COVID-19 pandemic, study sites for Omeros’ ongoing Phase 3 programs for narsoplimab in IgA nephropathy and aHUS are conducting trials in a manner consistent with local recommendations and/or regulations to maintain safety of study patients. As a result, at some sites, new patient enrollment has slowed while previously enrolled patients are continuing in the trials. We continue targeting data readout for the IgA nephropathy trial next year.

Updates regarding Omeros’ other development programs and platforms include the following:

First-in-human-enabling toxicology studies for the company’s MASP-3 inhibitor OMS906 are complete. Omeros is on track to file a clinical trial application this quarter and to begin dosing in the first part of the third quarter.

As part of the strategy for life-cycle management of the company’s complement franchise, Omeros continues to develop a longer-acting second generation antibody against MASP-2, which is targeted to enter the clinic in early 2022, to be followed by an orally available small molecule inhibitor against MASP-2 also under development.

Financial Results

For the first quarter of 2020, revenues, all related to sales of OMIDRIA, were $23.5 million, compared to $21.8 million for the same period in 2019 and a record-high $33.4 million for the fourth quarter of 2019. The decrease from the prior quarter reflects declining sales beginning in early March as a result of inventory utilization by ASCs and hospitals in anticipation of the COVID-19-related shutdown of elective surgical procedures, which occurred in mid-March. Sales of OMIDRIA to wholesalers were minimal in March. March typically accounts for about 45 percent of total first-quarter OMIDRIA revenues, and Omeros realized only one week of March revenues. In early May, a large number of states began re-opening ASCs and hospitals to cataract surgery, and facilities in at least 36 states have already initiated re-ordering of OMIDRIA from wholesalers

Total costs and expenses for the first quarter of 2020 were $47.2 million compared to $41.0 million for the comparable period in 2019. The increase was due to increased research and development and pre-commercialization marketing activities for narsoplimab.

For the three months ended March 31, 2020, Omeros reported a net loss of $29.0 million, or $0.53 per share, which included non-cash expenses of $6.4 million, or $0.12 per share. This compares to a net loss of $24.3 million, or $0.50 per share, which included non-cash expenses of $6.0 million, or $0.12 per share, for the comparable quarter in 2019.

As of March 31, 2020, the company had $54.0 million of cash, cash equivalents and short-term investments available for operations, a decrease $6.8 million from December 31, 2019. The company also has a line of credit, which permits borrowing up to the lesser of 85 percent of eligible accounts receivable and $50 million. As of March 31, 2020, the eligible accounts receivable balance was $24.1 million, and Omeros has not borrowed under this facility.

Conference Call Details

Omeros’ management will host a conference call to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 6549035. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 6549035.

To access the live or subsequently archived webcast of the conference call on the internet, go to the company’s website at www.omeros.com and select "Events" under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

On May 11, 2020 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, Acceleron Pharma, MAY 11, 2020, View Source [SID1234557505]).

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"Acceleron is off to a strong start in 2020, propelled by one of the most eventful time periods in the Company’s history," said Habib Dable, President and Chief Executive Officer of Acceleron. "The breadth and depth of our hematology program has been on full display, owing to the publication of the MEDALIST and BELIEVE Phase 3 luspatercept trial results in the prestigious New England Journal of Medicine and the recent FDA approval of REBLOZYL for the treatment of anemia in a population of patients with lower-risk MDS. We’re thrilled that this first-in-class erythroid maturation agent, having received approvals in two distinct indications over a span of just five months, is now available in the US to patients who have long needed a new option for treating their chronic anemias."

Added Mr. Dable: "Our scientific expertise in leveraging the therapeutic potential of the TGF-beta superfamily of proteins is delivering equally exciting results in pulmonary disease. With great enthusiasm, we announced that the PULSAR Phase 2 trial of sotatercept in patients with PAH met its primary and key secondary endpoints-underscoring our belief that sotatercept could eventually alter the current treatment paradigm. Following the results, sotatercept was granted Breakthrough Therapy designation by the FDA and PRIME designation by the EMA. We look forward to presenting topline results from PULSAR in a virtual meeting of the ATS as well as interactions with health authorities as we prepare for future Phase 3 development of sotatercept on our path to potential global registration."

Program Highlights

Hematology

REBLOZYL (luspatercept-aamt): Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
REBLOZYL is the first and only U.S. FDA approved erythroid maturation agent designed to promote red blood cell production through a novel mechanism. Luspatercept-aamt is being developed to treat anemia in patients with beta-thalassemia, MDS, and MF. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

In April, Acceleron and partner Bristol Myers Squibb announced the FDA approved REBLOZYL for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

The New England Journal of Medicine published results from the pivotal Phase 3 MEDALIST and BELIEVE trials.

In April, REBLOZYL received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the treatment of adult patients with:

transfusion-dependent anemia due to very low-, low- and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy; and

transfusion-dependent anemia associated with beta thalassemia

Bristol Myers Squibb expects to initiate the Phase 3 INDEPENDENCE trial in patients with MF on concomitant JAK 2 inhibitor therapy who require RBC transfusions by year-end 2020.

The BEYOND Phase 2 trial in adult patients with non-transfusion-dependent beta-thalassemia is ongoing, with topline results expected by year-end 2020 or early 2021.

Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with treatment-naïve, lower-risk MDS, with topline results expected in 2022.

Pulmonary

Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept (RAP-011) reversed pulmonary vessel muscularization and improved indicators of right heart failure.

In January, Acceleron reported that the PULSAR Phase 2 trial in patients with PAH met its primary and key secondary endpoints. Sotatercept was generally well tolerated in the trial and adverse events observed were generally consistent with previously published data on sotatercept in other diseases.

PULSAR Phase 2 topline results are expected to be presented in an American Thoracic Society (ATS) 2020 web-based session by the end of June 2020.

Acceleron plans to host a webcast and conference call for investors and analysts following the ATS 2020 virtual presentation to discuss highlights from the PULSAR trial results.

In April, sotatercept was granted Breakthrough Therapy designation by the FDA for the treatment of patients with PAH (World Health Organization Group 1).

The EMA recently granted Priority Medicines (PRIME) designation to sotatercept for the treatment of patients with PAH.

Enrollment is ongoing in the exploratory SPECTRA trial in patients with PAH, with preliminary results expected in 2020.

Financial Results

Cash Position – Cash, cash equivalents and investments as of March 31, 2020 were $415.6 million, as compared to $453.8 million as of December 31, 2019. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from REBLOZYL sales.

Revenue – Revenue for the first quarter of 2020 was $4.3 million, which includes $2.8 million of cost share revenue, and $1.5 million of royalty revenue from net U.S. sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.

Costs and Expenses – Total costs and expenses for the first quarter of 2020 were $55.9 million. This includes R&D expenses of $37.7 million and SG&A expenses of $18.3 million.

Net Loss – The Company’s net loss for the first quarter of 2020 was $50.9 million.

Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2020 financial results and provide an update on recent corporate activities on May 11, 2020, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron First Quarter 2020 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On May 11, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported its financial results for the first quarter ended March 31, 2020 (Press release, Castle Biosciences, MAY 11, 2020, View Source [SID1234557504]).

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"The Castle Biosciences team continued to perform at an exceptional level in the first quarter, with significant growth in revenue and DecisionDx-Melanoma test report volume," said Derek Maetzold, president and chief executive officer. "We are making investments in our business intended to put us in a position of strength as we continue to move through the current COVID-19 situation and execute on our strategy in the latter half of 2020 and 2021. This includes filling key positions and the initiation of clinical studies to drive additional evidence development for DecisionDx-Melanoma as well as to support the commercial launch of our two pipeline tests.

"With the recent publication of three peer-reviewed articles that document the clinical validity and impact of DecisionDx-SCC in patients diagnosed with high-risk cutaneous squamous cell carcinoma (SCC), we plan to launch this pipeline test in the third quarter of 2020. Our proprietary tests are used to inform important treatment plan decisions in early stage cancers and having the opportunity to expand our service to patients diagnosed with high-risk SCC is an important step. The planned launch of our gene expression profile test for suspicious pigmented lesions, our other near-term pipeline test, remains on track for the second half of 2020.

"The COVID-19 situation continues to evolve and brings along with it a high level of uncertainty surrounding potential future impacts. The pandemic has caused disruptions to patient flow and a significant reduction in biopsies and the number of patients with a diagnosis of cutaneous melanoma. During the first quarter, our business was not materially affected. However, from April 1, 2020 to May 6, 2020, orders for our lead product, DecisionDx-Melanoma, declined 43% from the same period in 2019, as a result of these patient flow disruptions due to COVID-19.

"In the midst of the COVID-19 pandemic, I would like to personally thank our employees for their dedication and tremendous efforts during these difficult times. We have implemented steps that focus on the health of our employees and their families and serving our customers. As a result, we have maintained access to our tests with our standard turnaround time, continuing to be, on average, less than five days from receipt of specimen to issuance of report, so that patients and their clinicians can incorporate our test results into their cancer treatment plan decisions without interruption. It is important to understand that we are able to meet the needs of the patients we serve, due to the dedication and effort of our team."

First Quarter Ended March 31, 2020, Financial Highlights

•Revenue was $17.4 million in the first quarter of 2020, compared to $8.7 million in the first quarter of 2019.

•Delivered 4,574 DecisionDx-Melanoma test reports in the first quarter of 2020, which represents 42% growth compared to the 3,232 reports delivered during the first quarter of 2019.
•New ordering clinicians for DecisionDx-Melanoma grew 43% in the first quarter, compared to the same period in 2019.
•Delivered 361 DecisionDx-UM test reports in the first quarter of 2020, which is consistent with the 360 reports during the first quarter of 2019.
•Gross margin in the first quarter of 2020 was 86%.
•Operating cash flow was $(0.3) million in the first quarter of 2020, compared to $1.3 million in the first quarter of 2019.

Cash and Cash Equivalents

As of March 31, 2020, the Company’s cash and cash equivalents totaled $98.7 million, and the outstanding principal balance on the Company’s bank term loan was $26.7 million.

COVID-19 Response and Impact

•The Company made adjustments to its laboratory operations in March designed to keep employees safe and provide uninterrupted access to its proprietary DecisionDx-Melanoma test and DecisionDx-UM test. The Company has maintained its specimen receipt to report turnaround time to on average less than 5 days.
•Based upon the analysis of the Company’s supply channel and inventory levels, the Company believes it has adequate access to reagents and consumables needed for testing patient samples and expects to continue providing normal turnaround times for delivery of its test reports.
•The Company observed a significant reduction in clinician orders of its DecisionDx-Melanoma test. Based upon results from its proprietary customer study, the Company believes that this is due to 1) an approximate 60% reduction in the number of patients seen, 2) the subsequent impact on reduced biopsies performed and 3) a reduction in melanomas diagnosed. A reduction in orders was first observed in the back half of March, and for the period of April 1 – May 6, 2020, the Company saw an overall decrease of 43% in Decision-Dx Melanoma orders, compared to the same period in 2019. The Company has observed a stabilization in the decline of orders, beginning in the second week of April.
•The DecisionDx-Melanoma dermatological clinician customers that participated in this proprietary customer study, indicated that they expect to expand office hours during the next two to three months, as they catch up on these rescheduled appointments, and that they intend to prioritize biopsies.
•In April 2020, the Company received $1.9 million in relief funds automatically allocated to Medicare providers under the Coronavirus Aid, Relief and Economic Security Act (CARES Act) and an advance payment of $8.3 million from the Centers for Medicare & Medicaid Services (CMS), which will be applied against future Medicare claims the Company submits for reimbursement later this year.

Cash and Cash Equivalents as of April 30, 2020

As of April 30, 2020, the Company had cash and cash equivalents of approximately $110 million. This amount is preliminary, subject to adjustment and based solely upon information available to the Company as of the date of this press release. It includes the $8.3 million advance payment received from CMS, which will increase the Company’s liabilities when recorded in the second quarter of 2020. This amount of cash and cash equivalents is not a comprehensive statement of the Company’s results of operations, liquidity or financial condition as of April 30, 2020, including with respect to the Company’s liabilities as of April 30, 2020, and has not been audited, reviewed or compiled by the Company’s independent registered public accounting firm. This amount is not intended to be indicative of expected cash and cash equivalents as of June 30, 2020, or other future fiscal periods. Accordingly, undue reliance should not be placed on this preliminary information, and it should be viewed in the context of all other available information regarding the Company’s results of operations, liquidity and financial condition.

Suspension of 2020 Revenue Guidance

Due to uncertainties regarding the duration and impact of the COVID-19 pandemic, the Company is suspending its previously announced annual revenue guidance for 2020.

Supplemental Revenue Information

Included in revenues for the quarters ended March 31, 2020 and 2019, were positive revenue adjustments related to tests delivered in prior periods of $3.2 million and $0.6 million, respectively. The additional positive revenue adjustments in the current year primarily relate to recognition of revenue for certain tests delivered in prior periods for which no revenue was recognizable originally but was recognized upon cash collection of payments for the tests in the current-year period.

First Quarter and Recent Clinical Evidence Updates

•Data from a systematic review and meta-analysis of the DecisionDx-Melanoma test was published in the Journal of the American Academy of Dermatology (JAAD). This meta-analysis included four study cohorts and demonstrated the strength and consistency of the test as an independent, significant predictor of recurrence and metastatic risk in patients with invasive cutaneous melanoma. Under the Strength of Recommendation Taxonomy (SORT) system, a systematic review and meta-analysis provide for the highest level of evidence for a prognostic biomarker (Level 1 evidence). The SORT system is used by the American Academy of Dermatology and other organizations to evaluate the quality, quantity and consistency of evidence supporting tests, such as DecisionDx-Melanoma.
•The development and validation of the Company’s cutaneous squamous cell carcinoma (SCC) prognostic test, DecisionDx-SCC, for patients diagnosed with high-risk cutaneous SCC, was recently published in JAAD. The results demonstrate that DecisionDx-SCC is the strongest, as well as an independent, predictor of metastatic risk relative to current SCC staging systems and can complement clinicopathologic risk factors to better stratify risk of metastasis in patients with high-risk SCC.
•Study data supporting a framework for integration of DecisionDx-SCC into risk-appropriate management of 300 high-risk cutaneous SCC patients (as defined by

NCCN), were recently published in Current Medical Research & Opinion (CMRO) and found combining DecisionDx-SCC class with American Joint Committee on Cancer (AJCC) T stage identified a group of 159 low-risk patients (Class 1, T1-T2) with a 7.5% rate of metastasis. Similarly, combining test results with Brigham and Women’s Hospital (BWH) staging identified 173 patients with a metastasis rate of 8.1%. Rates in both groups approached the rate observed for the general cutaneous SCC patient population. By comparison, Class 2B patients in the study had rates of metastasis surpassing 50%, regardless of the staging system with which it was combined, a rate that may warrant a high intensity plan with the NCCN management recommendations. Incorporation of DecisionDx-SCC results with T stage for these 300 patients with NCCN high risk features, more than 50% would have been recommended a low intensity management plan, while 34-39% would be recommended for a moderate intensity plan, and only 8% for a high intensity plan.
•Results from an intended use study conducted at the 2020 Winter Clinical Dermatology Conference involving 162 clinicians was also published as a companion article in CMRO. Using the established pre-test post-test vignette methodology, clinicians determined the treatment plan they would employ for patients with high-risk SCC. Treatment plan modalities included follow-up schedule, sentinel lymph node biopsy, nodal imaging, adjuvant radiation and adjuvant chemotherapy. Clinicians were then asked to determine the treatment plan with the addition of DecisionDx-SCC test results. The addition of a DecisionDx-SCC Class 1 test score resulted in more than a 60% reduction in treatment plan modality intensity, while a Decision-SCC Class 2B test score resulted in more than a 90% escalation in treatment plan modality intensity. Importantly, more than 95% of the changes were made in a risk appropriate manner within national guidelines for patient management.
•Another peer-reviewed manuscript demonstrating that DecisionDx-UM test results significantly impacted treatment plan recommendations for patients with uveal melanoma (UM) in a multicenter, prospective study was published in Melanoma Management. The multicenter CLEAR II study (Clinical Application of DecisionDx-UM Gene Expression Assay Results II) was designed to prospectively evaluate patterns of physician referral and metastatic surveillance regimens for UM patients who were tested with DecisionDx-UM as part of their diagnostic work up, and to compare management plans between DecisionDx-UM low-risk (Class 1) and DecisionDx-UM high-risk (Class 2) patients.

Conference Call and Webcast Details

Castle Biosciences will hold a conference call on Monday, May 11, 2020, at 4:30 p.m. Eastern time to discuss its first quarter 2020 results and provide a corporate update.

A live webcast of the conference call can be accessed here: View Source or via the webcast link on the Investor Relations page of the Company’s website (www.castlebiosciences.com). Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until June 1, 2020.

To access the live conference call via phone, please dial 877-282-2581 from the United States and Canada, or +1 470-495-9479 internationally, at least 10 minutes prior to the start of the call, using the conference ID 5699079.

There will be a brief Question & Answer session following management commentary.

On May 11, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported first quarter 2020 financial results and recent business highlights (Press release, Sangamo Therapeutics, MAY 11, 2020, View Source [SID1234557503]).

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"I’m proud of how the Sangamo team has worked together through the unusual circumstances presented by the COVID-19 pandemic," said Sandy Macrae, CEO of Sangamo. "While adhering to governmental workplace guidelines, we have sought to minimize disruptions to the progress of our research in the labs, our clinical trials, and our business development discussions. Importantly, Pfizer continues to target dosing a first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020, and we are working together to identify an opportunity to present additional data from the Phase 1/2 Alta Study. Our AAV manufacturing facility in Brisbane is expected to be operational by year end, and our cell therapy manufacturing units in Brisbane and in France in 2021. With the $350 million received from the recently closed Biogen collaboration, in addition to the $363 million in cash resources reported as of March 31st, we are moving ahead with significant balance sheet strength, and we are beginning to plan for resuming normal operations as shelter-in-place orders are lifted."

Recent Highlights
•Closed collaboration agreement with Biogen for development of gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases, receiving $225 million in stock sale proceeds and an additional $125 million upfront license fee.
•Executed a collaboration and exclusive global license agreement with UK cell conversion company Mogrify Ltd for Sangamo to develop allogeneic cell therapies from Mogrify’s proprietary induced pluripotent stem cells (iPSCs) cell conversion technology using Sangamo’s zinc finger protein (ZFP) gene-engineered chimeric antigen receptor regulatory T cell (CAR-Treg) platforms. This collaboration may have the potential to accelerate the development of scalable and accessible CAR-Treg cell therapies for the treatment of inflammatory and autoimmune diseases, diversifying Sangamo’s options and complementing current programs.
•Pfizer continues to target dosing the first patient in the Phase 3 hemophilia A gene therapy study in the second half of 2020. Details of the Phase 3 trial protocol were recently posted to clinicaltrials.gov, and Pfizer continues to recruit patients into the Phase 3 lead-in study. Pfizer and Sangamo are working together to identify an appropriate opportunity this year to provide the next clinical data update of results from the ongoing Phase 1/2 Alta Study.
•Successfully screened and enrolled the first several patients into the Phase 1/2 STAAR study evaluating ST-920 gene therapy for the treatment of Fabry disease and expect to initiate patient dosing at the earliest appropriate and safe opportunity in light of the COVID-19 pandemic.
•Appointed D. Mark McClung as Executive Vice President and Chief Business Officer leading commercial strategic planning, alliance management and corporate and business development.

First Quarter 2020 Financial Results
Cash, cash equivalents and marketable securities were $363.1 million as of March 31, 2020, compared to $384.3 million as of December 31, 2019. Since the end of the first quarter, the Company has received from Biogen $225.0 million for the issuance of Sangamo stock and a $125.0 million upfront license fee.

Consolidated net loss attributable to Sangamo for the first quarter ended March 31, 2020 was $42.9 million, or $0.37 per share, compared to a net loss of $42.2 million, or $0.41 per share, for the same period in 2019. Revenues

for the first quarter ended March 31, 2020 were $13.1 million, compared to $8.1 million for the same period in 2019.
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Non-GAAP operating expenses, which excludes stock-based compensation expense, were $52.0 million for the first quarter ended March 31, 2020, compared to $47.4 million for the same period in 2019. The increase in operating expenses reflects the Company’s headcount growth and facilities expansion to support the advancement of Sangamo’s therapeutic pipeline and manufacturing capabilities. These increases were partially offset by a decrease in clinical and manufacturing supply expenses.

Financial Guidance for 2020 reiterated (initially provided on February 28, 2020)
•On a GAAP basis, we continue to expect operating expenses in the range of $270 million to $285 million, including stock-based compensation expense of approximately $25 million.
•We continue to expect non-GAAP operating expenses, which excludes stock-based compensation expense, in the range of $245 million to $260 million.

Conference Call
Sangamo will host a conference call today, May 11, 2020, at 5:00 p.m. Eastern Time, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 6043504. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 6043504.