On August 13, 2020 Myriad Genetics, Inc. (NASDAQ: MYGN, "Myriad" or the "Company"), a global leader in molecular diagnostics and precision medicine, reported financial results for its fiscal fourth-quarter 2020 and provided an update on recent business activity (Press release, Myriad Genetics, AUG 13, 2020, View Source [SID1234563592]).

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"Following the substantial decline in test volumes at the end of Q3 and beginning of Q4 due to COVID-19 social distancing policies we saw a significant recovery in test volume trends throughout the quarter, with volumes in late June increasing to approximately 75% of the pre-pandemic level," said R. Bryan Riggsbee, interim president and CEO and CFO, Myriad Genetics. "As we look forward to fiscal year 2021, we are prepared to manage the business within whatever constraints that the COVID-19 pandemic imposes, and as such we will be investing in new capabilities such as telemedicine and direct-to-patient sample collection initiatives that will support the increase in test volumes above the Q4 levels towards pre-pandemic levels as quickly as possible. While we cannot predict the course of the pandemic or its full effect on our business we plan execute on several key business during fiscal year 2021 which should allow us to exit the year with growing momentum."

4Q20 Financial Highlights:

While test volumes were detrimentally impacted by the global pandemic, the company saw a significant recovery in overall test volume in the quarter. Averaged across our tests, volumes ended the quarter at approximately 75 percent of their pre-pandemic level.
Generated revenue of $93.2 million in the fiscal fourth-quarter 2020 and reduced total on-GAAP expenses by approximately $41 million from the prior quarter.
Total cash and cash equivalents and marketable securities increased sequentially in the fiscal fourth-quarter to $254.8 million.
Recent Business Highlights:

Received a final local coverage determination (LCD) for pharmacogenomic (PGx) testing by Palmetto GBA and CGS Administrators, LLC, two of the administrative contractors for the Centers for Medicare & Medicaid Services. The new LCD expands coverage for patients to all healthcare providers licensed and qualified to diagnose the condition and prescribe relevant medications (either independently or in an arrangement).
Published a new study for riskScore in the Journal of the American Medical Association Network Open demonstrating the ability of Myriad’s polygenic risk score to modify breast cancer risk stratification in women diagnosed with pathogenic mutations in common breast cancer genes.
Announced a new collaboration with OptraHEALTH to implement a cognitive ChatBOT named Gene which provides genetic and financial assistance information to prospective patients with the ability to answer over 500,000 health related questions.
Launched our proprietary AMPLIFY technology which increases maternal fetal fraction an average of 2.3 times and further increases the already market-leading accuracy of our Prequel non-invasive prenatal screening test.
Published a new study in Genetics in Medicine comprising over 93,000 patients which showed the challenges associated with ethnicity-based guidelines for carrier screening used by both the American College of Medical Genetics and the American College of Gynecology. In the study, following ethnicity-based guidelines from ACOG and ACMG would have resulted in 77 percent of carriers of severe genetic disease being missed.
Launched a new radiographic progression (RP) enhancement tool for the Vectra test report that is personalized based on the age, gender and adiposity of the patient. The new test report will provide individual risk of a patient’s risk of RP within one year.
Received favorable coverage decisions for Prolaris from four new commercial health plans including one of the top five national providers of health insurance. In aggregate the new health plans represent 26 million covered lives.
Received U.S. Food and Drug Administration (FDA) approval for the myChoice CDx test for use as a companion diagnostic by healthcare professionals to identify advanced ovarian cancer patients with positive homologous recombination deficiency status, who are eligible or may become eligible, for first-line maintenance treatment with Lynparza (olaparib) in combination with bevacizumab.
Financial Guidance
Given the continued unpredictability pertaining to the COVID-19 pandemic and the impact it has had on both customer behavior and our ability to market our tests to physician customers, the company continues to see a wide range of possible financial outcomes for fiscal year 2021. As a result, the company has decided to not provide fiscal year 2021 financial guidance.

Conference Call and Webcast
A conference call will be held today, Thursday, August 13, 2020, at 4:30 p.m. EDT to discuss Myriad’s financial results for the fiscal fourth-quarter and business developments. The dial-in number for domestic callers is 1-800-381-7839. International callers may dial 1-212-239-2905. All callers will be asked to reference reservation number 21966478. An archived replay of the call will be available for seven days by dialing (800) 633-8284 and entering the reservation number above. The conference call along with a slide presentation will be available through a live webcast at www.myriad.com.

On August 13, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the second quarter ended June 30, 2020 and provided a corporate update (Press release, Regulus, AUG 13, 2020, View Source [SID1234563591]).

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"We are pleased with the progress of our ADPKD program and the completion of dosing of the MAD clinical study for RGLS4326 in healthy volunteers," said Jay Hagan, CEO of Regulus. "We plan to initiate a Phase 1b study in patients with ADPKD to evaluate short-term treatment of RGLS4326 for safety, tolerability, pharmacokinetics, and changes in biomarkers of the disease. We have also recently completed additional non-clinical studies needed to address some of the FDA requirements to support studies of extended duration."

Second Quarter 2020 Corporate Highlights and Recent Updates

Appointment of Chief Scientific Officer: In August, Dr. Denis Drygin joined Regulus as its Chief Scientific Officer.

Program Updates

RGLS4326 for Autosomal Dominant Polycystic Kidney Disease: In July 2020, the Company completed dosing in the MAD clinical study for RGLS4326 in healthy volunteers. The Phase 1 MAD study is a randomized, double blind, placebo-controlled clinical trial evaluating three different dose levels of RGLS4326 for safety, tolerability, and pharmacokinetic properties. Top-line results showed that RGLS4326 is well-tolerated with no serious adverse events reported. Preliminary results suggest plasma exposure is dose proportional. In July 2020, the FDA granted Orphan Drug Designation to RGLS4326 for ADPKD.
The Company plans to initiate a Phase 1b open-label, short-term multiple dose study in patients with ADPKD in the second half of 2020. The study will evaluate RGLS4326 for safety, pharmacokinetics, and changes in levels of polycystin 1 (PC1) and polycystin 2 (PC2). Patients with ADPKD, due to the mutation in the PKD gene, have been reported to have low levels of PC1 and PC2. This study is designed to evaluate whether different dose levels of RGLS4326 can increase levels of PC1 and PC2 in ADPKD patients.

Advancement of Hepatitis B virus (HBV) Program: The Company has identified several microRNA targets that serve as host factors for the hepatitis B virus (HBV). Our lead compound directed to one of the host microRNAs has demonstrated nanomolar potency against HBV DNA replication and more than 95% reduction in Hepatitis B surface antigen in in vitro studies. Additionally, we have demonstrated reduction of HBV DNA, surface antigen and pgRNA in an in vivo efficacy model. We believe that targeting a host factor in the liver represents a unique mechanism of action for treatment of the virus compared to other programs in development and holds the potential for achieving a functional cure. We have nominated a development candidate and plan to commence IND-enabling activities.
Additional Research Updates: Cell therapies have been approved to treat a variety of hematological malignancies. Targeting solid tumors, however, has proven challenging for cell therapies due to the immune-suppressive effect of the tumor microenvironment (TME). The Company believes that ex vivo modulation of microRNA may enable cell therapy approaches to overcome the effects of the TME and address other challenges faced by cell therapies. The Company has demonstrated that targeting microRNA ex vivo can improve certain characteristics of engineered cells including an improved in vitro expansion, effector function, cytokine production, as well as resistance to exhaustion induced by tonic signaling. The Company is pursuing multiple applications of microRNA technology in a variety of cell therapies.
Second Quarter 2020 Financial Results

Cash Position: As of June 30, 2020, Regulus had $23.4 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $4.2 million and $7.4 million for the three and six months ended June 30, 2020, respectively, compared to $1.8 million and $7.8 million for the three and six months ended June 30, 2019, respectively. The aggregate increase for the three months ended June 30, 2020, as compared to the three months ended June 30, 2019, was driven by an increase in external development expenses, primarily attributable to the FDA lifting the partial clinical hold on the MAD study in December 2019 and completion of the dosing for the Phase 1 of the MAD study in July 2020. The aggregate decrease for the six months ended June 30, 2020, as compared to the six months ended June 30, 2019, was driven by a reduction in personnel and internal expenses, partially offset by an increase in external development expenses attributable to recommencement and completion of the dosing of the MAD study in July 2020.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.3 million and $4.7 million for the three and six months ended June 30, 2020, respectively, compared to $2.9 million and $6.4 million for the three and six months ended June 30, 2019, respectively. These amounts reflect personnel-related and ongoing general business operating costs. The decreases for the three and six months ended June 30, 2020, as compared to the three and six months ended June 30, 2019, are primarily attributable to continued cost reduction efforts subsequent to our corporate restructurings.

Revenue: Revenue was zero and less than $0.1 million for the three and six months ended June 30, 2020, respectively, compared to less than $0.1 million and $6.8 million for the three and six months ended June 30, 2019, respectively. Revenue recognized for the six months ended June 30, 2019 was attributable to the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $6.9 million, or $0.23 per share (basic and diluted), and $12.9 million, or $0.48 per share (basic and diluted), for the three and six months ended June 30, 2020, respectively, compared to $5.0 million, or $0.30 per share (basic and diluted), and $8.3 million, or $0.61 per share (basic and diluted), for the same periods in 2019.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The Company recently completed the multiple ascending dose study of RGLS4326 in healthy volunteers. Administration of RGLS4326 was well-tolerated with no serious adverse events reported. The Company is initiating a Phase 1b study in patients with ADPKD to evaluate short-term treatment of RGLS4326 for safety, tolerability, pharmacokinetics, and changes in biomarkers of the disease. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration beyond the current Phase 1b study until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from the recently completed additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.

On August 13, 2020 Repare Therapeutics Inc. ("Repare") (Nasdaq: RPTX), a leading precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics, reported financial results for the second quarter ended June 30, 2020, as well as recent operational highlights (Press release, Repare Therapeutics, AUG 13, 2020, View Source [SID1234563590]).

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"We set out in 2016 to become the leading precision oncology company focused on synthetic lethality in genomic instability and DNA damage repair, and have since built a strong pipeline of product candidates based upon our proprietary genome-wide, CRISPR enabled SNIPRx platform," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "With the successful completion of our initial public offering in June and our entry into a strategic collaboration with Bristol Myers Squibb in May, we have established a strong cash position to advance our pipeline. I am proud of our entire team and the remarkable progress they have made."

Operational Highlights:

Initiated a phase 1/2 clinical trial evaluating RP-3500 as a monotherapy and in combination with Pfizer’s PARPi, talazoparib. In July 2020, the Company received acceptance from the U.S. Food and Drug Administration (FDA) of an investigational new drug (IND) application and commenced dosing for the first patient in the Phase 1/2 clinical trial of RP-3500.
Completed initial public offering (IPO). In June 2020, Repare closed an upsized IPO of 12,650,000 of its common shares, including the exercise in full of the underwriters’ option to purchase up to an additional 1,650,000 common shares, at a public offering price of $20.00 per share. The net proceeds to Repare, after deducting underwriting commissions and offering expenses, were $232 million.
Bristol Myers Squibb collaboration. In May 2020, Repare entered into an exclusive, worldwide target discovery collaboration with Bristol Myers Squibb. Repare received an upfront payment from Bristol Myers Squibb of $65 million in Q2 2020, which included a $15 million equity investment in Repare in the form of a warrant that automatically exercised into 750,000 common shares at the public offering price of $20.00 per share upon IPO. In addition, Repare will be eligible to receive additional contingent payments of up to approximately $3 billion in the form of license fees, discovery, development, regulatory and sales-based milestones, in addition to royalty payments on net sales of each product commercialized by Bristol Myers Squibb.
Appointed new director. In June 2020, Repare appointed Ann D. Rhoads to its Board of Directors and as Chairperson of its Audit Committee.
Second Quarter 2020 Financial Results:

Cash and restricted cash: Cash and restricted cash as of June 30, 2020 were $370.1 million.
Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $9.0 million and $17.6 million for the three and six month periods ended June 30, 2020, respectively, as compared to $4.9 million and $8.6 million in the same periods in the prior year, respectively. Increases in R&D for the three and six month periods ended June 30, 2020 were primarily due to increases in development costs related to Repare’s RP-3500 and CCNE1-SL programs, as well as increases in personnel related expenses and certain other R&D expenses.
General and administrative (G&A) expenses: G&A expenses were $3.4 million and $5.4 million for the three and six month periods ended June 30, 2020, respectively, as compared to $1 million and $2.1 million in the same periods in the prior year, respectively. Increases in G&A for the three and six month periods ended June 30, 2020 were due to increases in payroll and personnel costs as well as increases in legal, professional and D&O insurance costs, which in turn increased as a result of our recent IPO.
Net loss: Net loss was $11.8 million, or $2.45 per share in the second quarter of 2020 and $24.4 million, or $7.56 per share, in the first half of 2020.
About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those patients most likely to achieve clinical benefit from resulting product candidates.

On August 13, 2020 IntelGenx Technologies Corp. (TSX V:IGX)(OTCQB:IGXT) (the "Company" or "IntelGenx") reported financial results for the second quarter ended June 30, 2020 (Press release, IntelGenx, AUG 13, 2020, View Source [SID1234563589]). All dollar amounts are expressed in U.S. currency, unless otherwise indicated, and results are reported in accordance with United States generally accepted accounting principles except where noted otherwise.

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2020 Second Quarter Financial Summary:

Revenue was $42,000, compared to $197,000 in the 2019 second quarter
Adjusted EBITDA was ($1.5 million), compared to ($2.1 million) in Q2-2019
Cash and short-term investments totaled $3.1 million as at June 30, 2020
Recent Developments:

Received its cannabis micro-processing license from Health Canada for its Montreal facility, in accordance with the Cannabis Act and Cannabis Regulations.
Announced that Health Canada authorized the sale of IntelGenx’s hand sanitizer product, both in liquid and gel formulations, which will be manufactured using excess capacity at the Company’s Health Canada-certified cGMP manufacturing facility in Montreal.
Announced that it had entered into a feasibility agreement with Cybin Corp., Canada’s premier mushroom life sciences company focused on advancing psychedelic and nutraceutical-based products derived from fungi, for the development of an orally-dissolving film for the delivery of pharmaceutical-grade psilocybin.
Announced that it had amended the exclusivity terms of its November 2018 license, development and supply agreement with Tilray, Inc. (NASDAQ:TLRY) ("Tilray") to allow for IntelGenx’s co-development and commercialization of CBD products with additional partners.
Announced a performance improvement program focused on generating near-term revenue, preserving the Company’s resources, extending its cash runway and accelerating the launch of RIZAPORT in Spain. The program also includes plans to respond to the U.S. Food and Drug Administration’s recent Complete Response Letter – which requested additional information, but no new bioequivalence study – related to IntelGenx’s resubmitted RIZAPORT new drug application ("NDA").
"This quarter was marked by the achievement of a very important milestone for IntelGenx: the receipt of our cannabis micro-processing license from Health Canada, which, in tandem with our amended agreement with Tilray, enables us to pursue additional partners to commercialize CBD-containing film products," said Dr. Horst G. Zerbe, CEO of IntelGenx. "In addition, we made significant progress on our performance improvement program by partnering with Cybin for the development of an orally-dissolving film for the delivery of pharmaceutical-grade psilocybin and receiving Health Canada authorization to sell our liquid and gel hand sanitizer formulations."

Financial Results:

Total revenues for the three-month period ended June 30, 2020 amounted to $42,000, a decrease of $155,000 compared to $197,000 for the three-month period ended June 30, 2019. The decrease is mainly attributable to a $155,000 decrease in Research and Development ("R&D") revenues.

Operating costs and expenses were $1.8 million for the second quarter of 2020, versus $2.5 million for the corresponding three-month period of 2019. The decrease for the three-month period ended June 30, 2020 is mainly attributable to a $580,000 decrease in R&D expenses and a $165,000 decrease in Selling, General and Administrative expenses.

For the second quarter of 2020, the Company had an operating loss of $1.8 million, compared to an operating loss of $2.3 million for the comparable period of 2019.

Net comprehensive loss for the three-month period ended June 30, 2020 was $1.3 million, or $0.01 per basic and diluted share, compared to net comprehensive loss of $2.5 million, or $0.03 per basic and diluted share, for the comparable period of 2019.

As at June 30, 2020, the Company’s cash and short-term investments totalled $3.1 million.

Conference Call Details:

IntelGenx will host a conference call to discuss these 2020 second quarter financial results today, Thursday, August 13, 2020, at 4:30 p.m. EDT. To participate in live call, please register here. The call will be also be webcast live and archived for twelve months at www.intelgenx.com.

On August 13, 2020 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported that the company will hold a conference call and live webcast on Thursday, August 27, 2020 at 4:30 p.m. Eastern Time (ET) to review its second quarter 2020 financial results and provide a business update (Press release, Ascendis Pharma, AUG 13, 2020, View Source [SID1234563588]).

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Conference Call Details

Date Thursday, August 27, 2020
Time 4:30 p.m. ET/1:30 p.m. Pacific Time
Dial In (U.S.) 844-290-3904
Dial In (International) 574-990-1036
Access Code 9987362
A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will be available on this website shortly after conclusion of the event for 30 days.