On October 29, 2020 OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three months ended September 30, 2020 (Press release, Opko Health, OCT 29, 2020, View Source;20.htm [SID1234569365]).

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Third Quarter Business Highlights

•BioReference Laboratories’ COVID-19 PCR testing volume increased 61% over the second quarter of 2020. During the third quarter, BioReference Laboratories (BRL) processed approximately 3.5 million COVID-19 PCR tests and currently has the capacity to process more than 70,000 tests per day. In addition, the laboratory performed approximately 300,000 COVID-19 serology tests to measure SARS-CoV-2 specific antibody levels with the capacity to process more than 400,000 serology tests per day.

BRL announced additional COVID-19 testing agreements for New York City schools through a continued collaboration with New York City, the Department of Health and Hospital Corporation, the Test & Trace Corp. and the Department of Education. Through these agreements, BRL is testing students, teachers and staff in nearly 1,000 public schools across New York. BRL continues to provide COVID-19 testing services to numerous states, cities, professional sports associations and healthcare organizations, as well as to over 600 drive-thru and retail pharmacy testing sites nationwide.

•Positive somatrogon Phase 3 topline results reported from crossover pediatric study. A global Phase 3 study of somatrogon administered once-weekly to children 3 to <18 years of age with growth hormone deficiency met its primary objective of improved treatment burden compared to daily injection of Genotropin as measured by the mean overall Life Interference total score after 12 weeks of treatment.

•Somatrogon global regulatory submissions: Pfizer remains on schedule with respect to its regulatory submissions for marketing approval of somatrogon for children with growth hormone deficiency in the U.S. in the fourth quarter of this year and in Europe and Japan in the first half of 2021.

•RAYALDEE has received marketing authorizations in seven European countries. Vifor Fresenius Medical Care Renal Pharma, OPKO’s commercial partner for RAYALDEE in Europe, has received marketing authorizations for RAYALDEE for the treatment of secondary hyperparathyroidism in adults with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency in the United Kingdom, Germany, Sweden, Norway, Ireland, Denmark and the Netherlands. Market authorizations from Spain, Portugal, Italy and Switzerland are still pending. Market launch of RAYALDEE in authorized countries is expected to begin next year.
•RAYALDEE total prescriptions reported by IQVIA increased 13% compared with the third quarter of 2019. Total prescriptions for the three months ended September 30, 2020 increased to approximately 16,700, compared with approximately 14,800 for the third quarter of 2019. During the third quarter of 2020, demand for RAYALDEE was impacted by challenges in onboarding new patients because of the COVID-19 pandemic.

•RAYALDEE Phase 2 clinical trial initiated in patients with mild-to-moderate COVID-19. OPKO initiated a Phase 2 clinical trial with RAYALDEE as a treatment for mild to moderate COVID-19. The trial, "A Randomized, Double-Blind Placebo-Controlled Study to Evaluate the Safety and Efficacy of RAYALDEE (calcifediol) Extended-release Capsules to Treat Symptomatic Patients Infected with SARS-CoV-2 (REsCue)," is expected to enroll approximately 160 subjects, many with stage 3 or 4 CKD who are at increased risk for developing more severe illness. The REsCue trial will randomize COVID-19 outpatients in a 1:1 ratio to 4 weeks of treatment with RAYALDEE or placebo, and 2 weeks of follow-up. Primary efficacy endpoints are raising and maintaining serum total 25-hydroxyvitamin D within the range of 50-100 ng/mL and time to resolution of COVID-19 symptoms. Numerous independent studies report a correlation between vitamin D status and COVID-19 risk and severity.

•GeneDx enters into agreement with Pediatrix Medical Group to offer neonatal genomic services. In August 2020, BRL’s GeneDx subsidiary announced an agreement with Pediatrix Medical Group, the nation’s leading provider of maternal-fetal, and pediatric medical and surgical subspecialty physician services, to offer state-of-the-art, next-generation genomic sequencing to support clinical diagnosis in neonatal intensive care units staffed by neonatologists affiliated with Pediatrix. The sequencing is designed to enhance diagnostic capabilities in order to lessen the impact of disease and to facilitate the development of novel precision medicine solutions for pediatric care.

•BioReference Laboratories launches best-in-class next-generation sequencing assay. In September 2020, BRL, along with its GenPath specialty oncology division, announced the launch of OnkoSight Advanced, a next-generation sequencing (NGS) assay that enables revolutionary deoxyribonucleic acid (DNA) mutational profiling of tumor samples. OnkoSight Advanced NGS testing provides targeted gene content that is aligned with the latest National Comprehensive Cancer Network and World Health Organization guideline recommendations to provide critical insight into many of the most common cancer types. Each OnkoSight Advanced panel includes key biomarkers – Tumor Mutation Burden and Tumor-Only Microsatellite, critical when profiling advanced-stage malignancies to guide potential immunotherapy.

Third Quarter Financial Results

•Net income for the third quarter of 2020 was $23.7 million, or $0.04 per diluted share, compared with a net loss of $62.0 million, or $0.11 per share, for the comparable period of 2019. Consolidated revenues for the third quarter of 2020 were $428.1 million compared with $228.8 million for the comparable period of 2019.
•Diagnostics: Revenue from services in the third quarter of 2020 was $392.5 million compared with $181.1 million in the prior-year period, primarily due to increased COVID-19 testing volumes, partially offset by reduced clinical and genomic test volumes related to the pandemic and lower clinical and genomic test reimbursement. In addition, the Company received a $10.0 million grant from the CARES Act in the third quarter. Total costs and expenses were $346.4 million in the third quarter of 2020 compared with $197.5 million in the third quarter of 2019. This increase represents higher volumes from both COVID-19 testing and from the core testing business. Operating income was $46.2 million in the third quarter of 2020 compared with an operating loss of $16.4 million in the prior-year period, an improvement of $62.5 million.
•Pharmaceuticals: Revenue from products in the third quarter of 2020 was $28.7 million compared with $26.2 million in the third quarter of 2019, with the increase primarily attributable to higher sales at OPKO Chile and an increase in net sales of RAYALDEE to $8.1 million in the third quarter of 2020 compared with $7.4 million in the prior-year period. Revenue from transfer of intellectual property was $6.8 million in the third quarter of 2020 compared with $20.7 million in the third quarter of 2019 reflecting a decrease in the amortization of payments received from Pfizer with respect to somatrogon. Total costs and expenses were $49.9 million in the third quarter of 2020 compared with $61.1 million in the prior-year period, with the decline primarily attributable to lower research and development expenses due to the completion of the pediatric human growth hormone Phase 3 trial. The operating loss was $14.4 million in the third quarter of 2020 compared with $14.2 million in the third quarter of 2019.
•Cash and equivalents: Cash, cash equivalents and marketable securities were $36.3 million as of September 30, 2020, which reflects the repayment, in full, of its line of credit with JP Morgan utilizing cash generated from operations of $63.0 million during the three months ended September 30, 2020. In addition, the Company has availability under its line of credit with JP Morgan of $64.7 million and an unutilized $100 million credit facility that provides access to incremental capital on a non-dilutive basis.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss third quarter financial results in greater detail during a conference call and live audio webcast at 4:30 p.m. Eastern time today, October 29, 2020. Participants are requested to pre-register for the conference call using the link here, or dialing (888) 869-1189 or (706) 643-5902 and using conference ID 4542807. Upon registering, participants will receive dial-in numbers, an event passcode and a unique registrant ID to gain immediate access to the call and bypass the live operator. Participants may pre-register at any time, including up to and after the start of the call.

To access the live call via webcast, please click on the link OPKO 3Q20 Results Conference Call. Individual investors and investment community professionals who do not plan to ask a question during the call’s Q&A session are encouraged to listen to the call via the webcast.

For those unable to listen to the live conference call, a replay can be accessed for a period of time on OPKO’s website at OPKO 3Q20 Results Conference Call. A telephone replay will be available beginning approximately two hours after the completion of the conference call. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and use conference ID 4542807.

On October 29, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported financial results for the third quarter ending September 30, 2020 and provided a corporate update (Press release, PTC Therapeutics, OCT 29, 2020, View Source [SID1234569364]).

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"I am very pleased by the rapid progress that we have made this quarter with the approval and strong U.S. launch of Evrysdi for spinal muscular atrophy and the continued growth across our global Duchenne muscular dystrophy franchise," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We have also made significant advances across our clinical pipeline with a registration-directed trial in mitochondrial epilepsy initiated this quarter and an additional registration-directed trial in Friedreich ataxia to initiate before year end. The mission at PTC has always been to bring therapies to patients with high unmet medical need through investment in innovative technologies and we believe we are well positioned to bring long-term value for all of our stakeholders."

Key Third Quarter and Other Corporate Updates

●In August 2020, the U.S. Food and Drug Administration (FDA) approved Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. In the early stages of launch, patients across a broad age range and SMA type have been treated. In October 2020, there were additional approvals in Brazil, Chile and Ukraine, further expanding the global reach of Evrysdi. Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation, and Roche.
●Also in August 2020, the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) filed by Roche for Evrysdi for the treatment of SMA. The EMA previously granted PRIME (PRIority MEdicines) designation to risdiplam for the treatment of SMA, providing a pathway for accelerated evaluation by the agency.
●The EMA’s acceptance of the MAA filed by Roche for Evrysdi for the treatment of SMA triggered a $15 million milestone payment to PTC and the first commercial sale of Evrysdi in the U.S. triggered a $20 million milestone payment to PTC. As a result, PTC recognized $35.0 million in collaboration revenue associated with Roche milestone events in the third quarter of 2020.
●In October 2020, Chugai Pharmaceutical, Co. Ltd, a member of the Roche group, announced that a new drug application for Evrysdi for the treatment of SMA was filed in Japan. Risdiplam received orphan drug designation from the Japan Ministry of Health, Labour and Welfare and the application is subject to a priority review. The filing in Japan triggered a $7.5 million milestone payment to PTC in the fourth quarter of 2020. Roche is executing an aggressive global regulatory strategy and now has over 20 additional applications filed for Evrysdi around the world.
●PTC has multiple aromatic L-amino acid decarboxylase (AADC) deficiency patient finding initiatives ongoing which continue to progress even amid COVID-19. PTC has expanded the number of
programs globally and in September 2020. PTC launched its newest program, PTC PINPOINT. The program offers testing at no charge to patients which can lead to an earlier diagnosis and treatment.

Third Quarter Clinical Updates

●Two-year data from Part 1 of the Evrysdi FIREFISH study were presented at the 25th International Annual Congress of the World Muscle Society (WMS). The data demonstrated that infants treated with Evrysdi continued to improve and achieve motor milestones.
●A registration-directed Phase 2/3 placebo-controlled trial to evaluate vatiquinone (PTC743) in children with mitochondrial epilepsy began enrollment in October 2020. This MIT-E trial is targeting the highly morbid and life-threatening symptom of refractory seizures in children with inherited mitochondrial disease. Vatiquinone, developed from PTC’s Bio-e platform, is an investigational oral small molecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways that underpin neurological disease pathology including epilepsy. The FDA has granted vatiquinone orphan drug designation and pediatric rare disease designation for mitochondrial epilepsy.
●The Phase 3 vatiquinone Friedreich ataxia trial (MOVE-FA) is expected to initiate in the fourth quarter of 2020 and the Phase 3 PTC923 phenylketonuria (PKU) trial is expected to initiate in mid-2021.
●Muscle biopsies have been completed on all 20 enrolled subjects in PTC’s TranslarnaTM (ataluren) dystrophin study (Study 045). The samples from these biopsies are in the process of being analyzed and PTC expects to report top-line results in the first quarter of 2021.
●A Phase 1 study of PTC518 in healthy volunteers is expected to begin enrollment this quarter. PTC518 is another candidate from PTC’s validated splicing platform and is being developed for the treatment of Huntington disease.
●The Phase 1 single ascending dose study for PTC857 has been completed and dosing in the multiple ascending dose study is expected to be completed by the end of 2020. PTC857 was developed from the Bio-e platform with the potential to address multiple CNS disorders, with the first planned indication being glucocerebrosidase (GBA) Parkinson’s disease.
●A Phase 2/3 clinical trial for PTC299 for COVID-19 (FITE19) continues to enroll patients. PTC is expecting Stage 1 of this two-stage trial to be completed by the end of 2020 and continues to anticipate reporting top-line results from both stages in the first half of 2021. Stage 1 of this study is being conducted in the U.S., Brazil, Spain, and Australia.
●Due to COVID-19 related delays, PTC expects the EMA’s Committee for Medicinal Products for Human Use final opinion for the AADC deficiency application in the first half of 2021. PTC expects the biologics license application (BLA) for AADC deficiency to be submitted to the FDA in the first half of 2021.

Financial Highlights

●Total revenue was $118.4 million for the third quarter of 2020, compared to $71.4 million for the third quarter of 2019, a 66% year-over-year increase, Total revenue includes net product revenue of $82.7 million and collaboration and royalty revenue of $35.7 million in the third quarter of 2020.
●Total net product revenue across the commercial portfolio was $82.7 million for the third quarter of 2020, a 16% year-over-year increase.
●The Duchenne muscular dystrophy franchise continued to show strong performance in the third quarter with a 15% year-over-year increase. Emflaza continued its momentum from the second quarter of 2020 into the third quarter of 2020 driven by new patient prescriptions and ongoing
operational improvements in PTC’s commercial business. New Duchenne patients continue to be identified in Europe, LATAM and other key markets for Translarna.
●Translarna net product revenue was $43.4 million for the third quarter of 2020, compared to $48.3 million for the third quarter of 2019, a 10% year-over-year decrease. Revenue for the third quarter of 2020 was impacted by the timing of a group purchase order from Brazil, which is the primary driver for the year-over-year decrease compared to the third quarter of 2019.
●In October, PTC entered into a purchase agreement with the Brazil Ministry of Health for Translarna. The initial shipment was received by the Brazil Ministry of Health in October 2020 with the additional shipment expected in the first half of 2021.
●Emflaza net product revenue was $38.5 million for the third quarter of 2020, compared to $22.9 million for the third quarter of 2019. Growth in net product revenue was driven by new patient prescriptions and continued operational improvements and efficiencies in the commercial business.
●Roche reported initial Evrysdi August and September sales of approximately CHF 8 million. The acceptance of the MAA filed by Roche for Evrysdi for the treatment of SMA triggered a $15 million milestone payment to PTC and the first commercial sale of Evrysdi in the U.S. triggered a $20 million milestone payment to PTC. As a result, PTC recognized $35.0 million in collaboration revenue associated with Roche milestone events in the third quarter of 2020.
●Based on U.S. GAAP (Generally Accepted Accounting Principles), research and development (R&D) expenses were $93.0 million for the third quarter of 2020, compared to $63.1 million for the third quarter of 2019. The increase in R&D expenses reflects costs associated with advancing the gene therapy and Bio-e platforms, increased investment in research programs, and advancement of the clinical pipeline.
●Non-GAAP R&D expenses were $83.8 million for the third quarter of 2020, excluding $9.2 million in non-cash, stock-based compensation expense, compared to $58.1 million for the third quarter of 2019, excluding $5.0 million in non-cash, stock-based compensation expense.
●GAAP selling, general and administrative (SG&A) expenses were $57.8 million for the third quarter of 2020, compared to $49.3 million for the third quarter of 2019. The increase reflects continued investment to support PTC’s commercial activities including the expanding commercial portfolio.
●Non-GAAP SG&A expenses were $50.2 million for the third quarter of 2020, excluding $7.6 million in non-cash, stock-based compensation expense, compared to $43.8 million for the third quarter of 2019, excluding $5.5 million in non-cash, stock-based compensation expense.
●Change in the fair value of deferred and contingent consideration was $8.4 million for the third quarter of 2020, compared to $9.5 million for the third quarter of 2019. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
●Net loss was $69.7 million for the third quarter of 2020, compared to net loss of $60.0 million for the third quarter of 2019.
●Cash, cash equivalents and marketable securities were $1,141.0 million as of September 30, 2020, compared to $686.6 million as of December 31, 2019. The cash, cash equivalents and marketable securities balance as of September 30, 2020 includes $650.0 million in consideration received related to the July 17, 2020 Royalty Purchase Agreement between PTC and RPI 2019 Intermediate Finance Trust ("RPI").
●Shares issued and outstanding as of September 30, 2020 were 67,809,560.

Non-GAAP Financial Measures

In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.

Conference Call and Webcast

PTC will host a conference call to discuss the third quarter of 2020 operational and financial today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 7096445. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On October 29, 2020 Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC, Nasdaq:CYCCP) ("Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported the appointment of Mark Kirschbaum, M.D. as Senior Vice President & Chief Medical Officer (CMO) (Press release, Cyclacel, OCT 29, 2020, View Source [SID1234569363]). Dr. Kirschbaum is a highly experienced hematologist/oncologist with over 30 years of experience in molecular medicine, new drug development, clinical trial design and patient care. He has management experience in both academic research and clinical and pharmaceutical settings. As CMO, he will be responsible for advancing Cyclacel’s pipeline and will lead clinical strategy, patient safety, and medical affairs.

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"We are delighted to welcome Mark to the Cyclacel team," said Spiro Rombotis, Cyclacel’s President & Chief Executive Officer. "Recent data with fadraciclib, our CDK2/9 inhibitor, and CYC140, our PLK1 inhibitor, support further clinical development of these agents in both liquid and solid cancers. Mark’s extensive hematology and oncology experience in clinical practice, experimental therapeutics and industry drug development will be essential as we advance these and our other clinical development programs with the aim of helping patients with unmet medical needs."

"Cyclacel’s biomarker-driven approach to drug development has produced a growing and diversified clinical pipeline with the potential to target a broad range of malignancies," said Dr. Kirschbaum. "I am excited to join the Cyclacel team at this point in its evolution to help build an innovative pipeline addressing the rising problem of cancer resistance and to achieve our clinical milestones."

Dr. Kirschbaum will report to Spiro Rombotis, President and Chief Executive Officer. He will be based in the Company’s Berkeley Heights, NJ office.

Most recently, Dr. Kirschbaum served as Vice President, Hematology/ Oncology at ArQule Inc., (recently acquired by Merck & Co.) where he managed the development of their BTK inhibitor ARQ531 for hematological indications, including CLL. Prior to ArQule, he was Senior Medical Director with global clinical development responsibilities at Daiichi-Sankyo, Taiho Pharmaceuticals and BeiGene, USA, where he led the clinical development of novel compounds including inhibitors of EZH2/1, HSP-90, HER2/3 and BTK in various solid tumors and hematological malignancies.

Before working in the biopharmaceutical industry, Dr. Kirschbaum served as Professor of Medicine, Director of Experimental Therapeutics, Hematology at the Monter Cancer Center/NSLIJHS; Professor of Medicine, Director Hematologic Malignancies at Penn State, Hershey Cancer Center, Director of Experimental Therapeutics, Nevada Cancer Institute, and Director, New Drug Development at the City of Hope National Cancer Center, and Attending Senior Physician, Department of Hematology and Department of Bone Marrow Transplantation, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel.

He has earned a B.A. from Yeshiva University in New York and his M.D. from SUNY–Health Sciences Center in Brooklyn. He did his Residency in Internal Medicine at Kings County Hospital Center in New York. He also held a Research Fellowship in Oncology at Fred Hutchinson Cancer Research Center in Seattle and worked as a physician scientist at Hadassah University Hospital and the Weizmann Institute of Science in Israel.

Cyclacel also announced that the Compensation Committee of its Board of Directors authorized the grant to Dr. Kirschbaum of non-qualified stock options to purchase up to 120,000 shares of the Company’s common stock, effective as of the first day of his employment as an inducement to Dr. Kirschbaum to commence employment with Cyclacel. The award was granted under Cyclacel’s 2020 Inducement Equity Incentive Plan which Cyclacel’s Board of Directors adopted to facilitate the granting of equity awards to new employees in accordance with NASDAQ Listing Rule 5635(c)(4).

The inducement grant is exercisable at a price of $3.77 per share, which is the closing price per share of Cyclacel’s common stock as reported by NASDAQ on October 23, 2020. The stock option shall vest over three years, with one third of the award vesting on October 23, 2021, and the remainder vesting ratably at the end of each subsequent month thereafter, subject to Dr. Kirschbaum’s continued employment with Cyclacel through each applicable vesting date. The option has a ten-year term and is subject to the terms and conditions of a stock option agreement.

On October 29, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the third quarter ended September 30, 2020 and revised upward its full-year 2020 financial guidance for product revenue (Press release, Vertex Pharmaceuticals, OCT 29, 2020, View Source [SID1234569362]).

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"This has been another very strong quarter for Vertex with execution across our CF business and continued earnings and revenue growth," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "We are pleased with our progress toward treating all people with cystic fibrosis highlighted by several recent milestones: the early approval and encouraging start of the launch of KAFTRIO in the EU, positive TRIKAFTA data in children ages 6-11, and continued expansion of our medicines’ labels as with our recent approval of KALYDECO for infants as young as 4 months of age."
"As we extend our leadership in CF, we are also advancing a broad pipeline of innovative therapies," continued Dr. Kewalramani. "Our R&D strategy contemplates the high-risk nature of drug development and therefore includes a portfolio approach to each of our disease areas of interest. In AATD, while disappointed by the VX-814 outcome, we look forward to the VX-864 Phase 2 proof-of-concept data in the first half of 2021. Our pipeline spans multiple diseases, and multiple important clinical readouts are expected from now through the end of 2021, each of which we expect will hold transformative potential for patients and further growth for Vertex."

Product revenues increased 62% compared to the third quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S. and the uptake of our medicines outside the U.S. following the completion of several significant reimbursement agreements.
GAAP and non-GAAP net income increased compared to the third quarter of 2019, largely driven by strong growth in total product revenues.
Cash, cash equivalents and marketable securities as of September 30, 2020 were $6.2 billion, an increase of approximately $2.3 billion compared to $3.8 billion as of December 31, 2019 driven by strong revenue and profitability.
Combined GAAP R&D and SG&A expenses decreased compared to the third quarter of 2019 due to a decrease in collaboration payments.
Combined Non-GAAP R&D and SG&A expenses increased compared to the third quarter of 2019, primarily due to the incremental investment to support the global use of Vertex’s medicines and the expansion of Vertex’s pipeline in CF and other disease areas.
GAAP and Non-GAAP income taxes increased compared to the third quarter of 2019 primarily due to Vertex’s increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.

Full-Year 2020 Financial Guidance
Vertex today revised upward its guidance for full-year 2020 product revenues. The company also adjusted its expectation for combined GAAP R&D and SG&A expenses and non-GAAP effective tax rate. Vertex’s guidance is summarized below:
Key Business Highlights:

Cystic Fibrosis (CF) R&D pipeline:
Vertex expects to increase the number of CF patients eligible to take our medicines and thereby continue to grow our CF business. Important progress has been made in supporting the extension of the eligible patient population and expansion to additional geographies and age groups.

TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)
•The European Commission granted marketing authorization for KAFTRIO to treat people with CF ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations.
•The European Medicines Agency (EMA) validated a Type II Variation Marketing Authorization Application (MAA) for KAFTRIO that will support future indication expansion of the EU label to people with CF who have one copy of the F508del mutation.
•Vertex reported positive Phase 3 data for the elexacaftor/tezacaftor/ivacaftor triple combination in children with CF ages 6-11 who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. Vertex expects to file a supplemental New Drug Application (sNDA) with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020.
•The FDA accepted three sNDAs for TRIKAFTA, SYMDEKO and KALYDECO. These regulatory submissions are intended to expand the labels of these drugs to include additional people with CF who have rare CFTR mutations.
•Vertex is initiating a Phase 3 study for the elexacaftor/tezacaftor/ivacaftor triple combination in children with CF ages 2-5 who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.
SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)
•Vertex announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of SYMKEVI for the treatment of children with CF ages 6-11 with two F508del mutations or one F508del mutation and certain residual function mutations.

KALYDECO (ivacaftor)
•The FDA approved KALYDECO for use in infants with CF ages four months to less than six months old who have at least one mutation that is responsive to KALYDECO.
•Vertex announced that the EMA’s CHMP adopted a positive opinion for the label extension of KALYDECO for the treatment of infants with CF ages 4 months to less than 6 months who have at least one mutation that is responsive to KALYDECO.

Genetic therapies
•Vertex and Moderna established a new collaboration aimed at the discovery and development of lipid nanoparticles (LNPs) and mRNAs that can deliver gene-editing therapies to cells in the lung for the treatment of CF.

R&D pipeline outside of CF:
Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

Beta Thalassemia and Sickle Cell Disease:
•Vertex and its partner CRISPR Therapeutics are evaluating the use of an ex-vivo CRISPR gene-edited therapy for the treatment of transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD). This approach aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin in red blood cells, which has the potential to reduce or eliminate symptoms associated with disease.
•Vertex and CRISPR Therapeutics previously announced that, as of June, seven patients had been dosed across its two Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 and presented data at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress from two TDT patients and one SCD patient. Additional patients have been enrolled and dosed in both TDT and
SCD studies and the company expects to report clinical data from more patients treated with CTX001 in addition to data from patients with longer follow-up in the fourth quarter.
•The EMA granted Priority Medicines (PRIME) designation to CTX001 for the treatment of severe SCD. CTX001 has also been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA and Orphan Drug Designation from the European Commission for both TDT and SCD.

Alpha-1 Antitrypsin (AAT) Deficiency:
•Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency.
•Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021.
•In October, Vertex discontinued development of VX-814 based on the safety and pharmacokinetic profile of VX-814 observed to date in the Phase 2 clinical study.

APOL1-mediated Kidney Diseases:
•Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS).
•Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147. Data from this study is expected in 2021.

Type 1 Diabetes (T1D):
•Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
•Vertex has completed the required enabling nonclinical studies and manufacturing work to support the submission of an Investigational New Drug (IND) application to the U.S. FDA for the islet cells alone program in the fourth quarter of 2020.

On October 29, 2020 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, reported a business update and announced financial results for the third quarter ended September 30, 2020 (Press release, Corvus Pharmaceuticals, OCT 29, 2020, View Source [SID1234569361]).

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"We have several important milestones for our cancer and COVID-19 programs in the coming months," said Richard A. Miller, M.D., president and chief executive officer of Corvus. "For our lead program, ciforadenant combined with atezolizumab in renal cell cancer (RCC), we are on track to meet with FDA in December to finalize the design of a Phase 3, biomarker driven clinical trial focused on RCC patients that are positive for the Adenosine Gene Signature. The data on the signature, which has been confirmed independently, suggests that positive patients may be most likely to benefit from treatment with ciforadenant. We are also encouraged that our ITK inhibitor, CPI-818, has shown activity in patients with advanced peripheral T cell lymphoma, and we look forward to sharing updated data at the ASH (Free ASH Whitepaper) meeting in December."

"For CPI-006, we continued to enroll patients in our Phase 1 cancer study and in our recently launched Phase 1 study in patients with COVID-19. The initial results in COVID-19 patients have been encouraging and aligned with our expectations based on CPI-006’s unique mechanism of B cell activation and subsequent antibody and memory B cell production. This includes the generation of a polyclonal immune response to the SARS-CoV-2 virus induced by relatively low doses of CPI-006, which may offer advantages over passive administration of monoclonal antibodies. We will present the latest data on the COVID-19 program at the SITC (Free SITC Whitepaper) meeting in November, and based on the positive results to-date, plan to initiate a larger registrational study before the end of the year."

"On the corporate development front, in October we co-founded Angel Pharmaceuticals, a biopharmaceutical company based in China. Angel expands our pipeline into the large and growing Chinese healthcare market and provides the potential to accelerate our product development on a global basis. In addition, Corvus will initially retain a 49.7% ownership position in the company."

Recent Achievements

CPI-006: Anti-CD73 Antibody with Immunomodulatory Activity

Initiated an open-label, Phase 1 study of CPI-006 in COVID-19 patients with mild to moderate symptoms that require hospitalization and reported initial data that supports its potential as a novel immunotherapy approach for these patients. This includes the inducement of sustained high titers of SARS-CoV-2 specific antibodies and increased levels of memory B cells, with a dose-response effect seen in the first two cohorts with prolonged high titers of antibodies observed out to 56 days.

Published results from initial cohorts of COVID-19 study and data characterizing the novel immunotherapy approach with CPI-006 online at medRxiv.org. The preclinical data in the manuscript demonstrate that administration of CPI-006 resulted in B cell activation, antibody secretion and induction of memory B and T cells. The clinical data in the manuscript is consistent with laboratory studies and reflected high titers of IgG and IgM anti-SARS-CoV-2 antibodies.

Completed planned enrollment in each of the study’s four cohorts (0.3, 1.0, 3.0 and 5.0 mg/kg), with additional enrollments ongoing in order to expand certain cohorts. Additional data from the study, including results from all four cohorts, will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in November.

Completed enrollment in three dose escalation arms of the CPI-006 Phase 1/1b cancer clinical trial: monotherapy, combination with ciforadenant and combination with pembrolizumab and we continue to enroll the triplet combination dose escalation arm with ciforadenant and pembrolizumab.
CPI-818: A small molecule ITK inhibitor

Continued follow-up on patients enrolled in the CPI-818 Phase 1/1b clinical trial, with a focus on the seven patients with peripheral T-cell lymphoma (PTCL). To-date, there have been two responses in this group – one patient who previously failed chemotherapy and high dose chemotherapy with autologous bone marrow transplantation that achieved a complete response and completed one year of treatment; and one patient who failed multiple prior therapies that achieved a partial response.
Co-Founding of Angel Pharmaceuticals in China

Announced the co-founding of Angel Pharmaceuticals in China to create clinical study synergies and accelerate development timelines for the Company’s pipeline (ciforadenant, CPI-006 and CPI-818) while retaining 49.7% ownership of Angel.
Anticipated Future Events

The Company will present Phase 1 study results with CPI-006 in COVID-19 patients at the SITC (Free SITC Whitepaper) annual meeting, which is being held virtually on November 9-14, 2020.

The SITC (Free SITC Whitepaper) poster presentation will be available on November 9 at 8:00 am ET on the SITC (Free SITC Whitepaper) meeting website.
Title:
Immunotherapy with B cell activating antibody CPI-006 in patients (pts) with mild to moderate COVID-19 stimulates anti-SARS-CoV-2 antibody response, memory B cells and memory T effector cells
Poster #: 325
Lead Author: Gerard J. Criner, MD, Temple University Hospital
Category: In-Progress Clinical Trials
Based on interim data from the Phase 1 study, and assuming the remainder of the data in the study supports it, begin a pivotal, randomized, double blind study of CPI-006 in hospitalized COVID-19 patients in December 2020, with results expected to be available around mid-2021.

On track to meet with the U.S. Food and Drug Administration (FDA) in December 2020 to obtain feedback on the Company’s proposed pivotal trial for ciforadenant in patients with refractory renal cell carcinoma. The proposed pivotal trial will utilize the Company’s Adenosine Gene Signature biomarker to select patients who may be most likely to benefit from treatment with ciforadenant plus atezolizumab.

Present updated clinical data from the CPI-818 Phase 1/1b clinical trial at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2020.
Financial Results

At September 30, 2020, Corvus had cash, cash equivalents and marketable securities totaling $51.4 million, as compared to cash, cash equivalents and marketable securities of $78.0 million at December 31, 2019.

Research and development expenses for the three months ended September 30, 2020 totaled $6.6 million compared to $9.0 million for the same period in 2019. The decrease of $2.4 million was primarily due to a $0.8 million decrease in CPI-818 drug manufacturing costs, a $0.8 million decrease in outside research service costs and a $0.5 million decrease in personnel costs.

General and administrative expenses for the three months ended September 30, 2020 totaled $3.2 million dollars compared to $2.5 million for the same period in 2019. The increase of $0.7 million primarily consisted of an increase in professional service costs.

The net loss for the three months ended September 30, 2020 was $9.8 million, compared to a net loss of $11.0 million for the same period in 2019. Total stock compensation expense for the three months ended September 30, 2020 was $1.3 million compared to $1.7 million for the same period in 2019.