Cytokinetics Reports Third Quarter 2019 Financial Results

On October 31, 2019 Cytokinetics, Incorporated (Nasdaq:CYTK) reported financial results for the third quarter of 2019 (Press release, Cytokinetics, OCT 31, 2019, View Source [SID1234550117]). Net loss for the third quarter was $29.6 million, or $0.50 per share, compared to net loss for the third quarter of 2018 of $22.0 million, or $0.40 per share. Cash, cash equivalents and investments totaled $166.0 million at September 30, 2019.

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"In the third quarter of 2019, we achieved major milestones in both our cardiovascular and neuromuscular programs representing progress on key priorities across our pipeline of muscle-directed therapies," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "Completion of enrollment of GALACTIC-HF represents a significant step forward towards the completion of this important trial which holds promise for advancing the treatment of millions of patients suffering from heart failure. Additionally, recently presented Phase 1 data for CK-274 reaffirms its next-generation profile and supports advancing this program into a Phase 2 trial in patients with obstructive hypertrophic cardiomyopathy this year. Furthermore, additional analyses of results from FORTITUDE-ALS, as well as ongoing regulatory interactions, provide support for a potential Phase 3 trial of reldesemtiv in patients with ALS next year. We believe this progress positions us well to leverage our leadership in muscle pharmacology to benefit a wide array of patients suffering from varied diseases of muscle dysfunction."

Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Completed patient enrollment in GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil. GALACTIC-HF enrolled over 8,200 patients in 35 countries. We expect GALACTIC-HF to continue throughout 2019 and the second planned interim analysis of GALACTIC-HF to occur in the first quarter of 2020.

Continued conduct of METEORIC-HF (Multicenter Exercise Tolerance Evaluation of Omecamtiv Mecarbil Related to Increased Contractility in Heart Failure), the second Phase 3 trial of omecamtiv mecarbil. METEORIC-HF is a randomized, placebo-controlled, double-blind, parallel group, multicenter clinical trial designed to evaluate the effect of treatment with omecamtiv mecarbil compared to placebo on exercise capacity as determined by cardiopulmonary exercise testing (CPET) following 20 weeks of treatment. We expect to continue enrollment of METEORIC-HF throughout 2019.
AMG 594 (cardiac troponin activator)

Continued conduct of the Phase 1 study of AMG 594 to assess its safety, tolerability, pharmacokinetics and potential to increase cardiac function in healthy volunteers. AMG 594 is a novel, selective, oral, small molecule cardiac troponin activator, discovered under our joint research program with Amgen. This Phase 1 study is being conducted by Amgen in collaboration with Cytokinetics. We expect the conduct of this study to continue throughout 2019.
CK-3773274 (CK-274, cardiac myosin inhibitor)

Presented data from the Phase 1 study of CK-274 at the Heart Failure Society of America’s 23rd Annual Scientific Meeting in Philadelphia. The study met its primary and secondary objectives to assess the safety and tolerability of single and multiple oral doses of CK-274, describe the pharmacokinetics of CK-274 and its pharmacodynamic effects as measured by echocardiography, as well as to characterize the PK/PD relationship with regards to cardiac function. These data support the advancement of CK-274 into a Phase 2 clinical trial in patients with obstructive hypertrophic cardiomyopathy (HCM) which is expected to begin in Q4 2019.

Presented preclinical data at the American Heart Association’s Basic Cardiovascular Sciences Scientific Sessions in Boston demonstrating that CK-274 produces exposure related effects on cardiac contractility in healthy animals and mouse models of HCM and support the therapeutic hypothesis relating to onset of action and reversibility.
Skeletal Muscle Program

reldesemtiv (next-generation, fast skeletal muscle troponin activator (FSTA))

Presented post-hoc analyses from FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS), at the 2019 Northeast Amyotrophic Lateral Sclerosis (NEALS) Meeting in Clearwater Beach, FL. The analyses demonstrated that, in the combined middle and faster progressing tertiles of patients, the decline in the ALSFRS-R total score from baseline to week 12 in patients who received any dose of reldesemtiv was significantly smaller than the decline on placebo, while no significant difference between reldesemtiv and placebo was observed in slower progressing patients.

Held regulatory interactions and conducted feasibility and other planning activities in preparation for the potential advancement of reldesemtiv to a Phase 3 trial in patients in ALS in 2020.

Received European Orphan Designation for reldesemtiv for the potential treatment of spinal muscular atrophy (SMA) by the European Medicines Agency (EMA).
Pre-Clinical Development and Ongoing Research

Announced the publication of "Fast Skeletal Muscle Troponin Activator CK-2066260 Increases Fatigue Resistance by Reducing the Energetic Cost of Muscle Contraction," in The Journal of Physiology, demonstrating that a FSTA can decrease skeletal muscle fatigue by increasing the metabolic efficiency of muscle contraction. CK-2066260 is a preclinical FSTA tool compound.

Continued pre-clinical development of CK-3762601 (CK-601), a next-generation FSTA, under our collaboration with Astellas.

Continued research in collaboration with Astellas directed to the discovery of next-generation skeletal muscle activators; Astellas is sponsoring Cytokinetics’ research activities through 2019.

Continued independent research activities directed to our other muscle biology research programs.
Corporate

We and Astellas have agreed in principle to revise the terms of our collaboration agreement so that Cytokinetics would have the exclusive right to develop and commercialize all FSTAs, including reldesemtiv and CK-601. Astellas’ future contributions would be to provide partial co-funding for certain Phase 3 clinical trial costs for reldesemtiv in ALS and to provide other in-kind support. In exchange, Astellas would receive a low- to mid- single digit royalty on reldesemtiv to be payable by Cytokinetics. We have also agreed in principle to extend our joint research program for another year with Astellas sponsoring research at Cytokinetics through 2020.

The above agreements in principle are non-binding and contingent upon our finalizing amendments to our collaboration agreement and, absent such agreement, the terms of the existing agreement remain in place.

Announced the continuation of our partnership with Cure SMA to increase education, awareness, public policy and fundraising for SMA. The partnership includes support for several of Cure SMA’s national initiatives as well as local community events.

Announced a call for proposals for the second annual Cytokinetics Communications Fellowship Grant program. The program provides $100,000 in grants to five selected patient advocacy organizations serving the ALS, heart failure, HCM, or SMA communities, and is intended to support increased capacity in communications, awareness building and community engagement.
Financials

Revenues for the three and nine months ended September 30, 2019 were $6.1 million and $21.7 million, respectively, compared to $10.6 million and $22.1 million for the corresponding periods in 2018. The decrease in revenues for the three and nine months ended September 30, 2019 was due primarily to the winding down of FORTITUDE-ALS in addition to a lack of license revenue in 2019. License revenues in the third quarter and first nine months of 2018 were related to the Phase 2 trial of reldesemtiv in spinal muscular atrophy completed in 2018.

Research and development expenses for the three and nine months ended September 30, 2019 were $20.2 million and $67.8 million, respectively compared to $21.4 million and $65.9 million for the same periods in 2018, respectively. The changes were primarily due to reduced spending for reldesemtiv as well as tirasemtiv following suspension of development of tirasemtiv in late 2017 offset by increased spending related to METEORIC-HF and the development of CK-274.

General and administrative expenses for the three and nine months ended September 30, 2019 increased to $9.8 million and $29.0 million, respectively, from $7.2 million and $23.7 million for the same periods in 2018, respectively, due primarily to an increase in outside legal counsel and personnel related costs including stock-based compensation.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s third quarter 2019 results via a webcast and conference call today at 4:30 PM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 9440838.

An archived replay of the webcast will be available via Cytokinetics’ website until November 7, 2019. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 9440838 from October 31, 2019 at 7:30 PM Eastern Time until November 7, 2019.

Audentes Therapeutics to Release Third Quarter 2019 Financial Results and Provide Corporate Update on Thursday, November 7, 2019

On October 31, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, reported that it will host a conference call and webcast to report its third quarter 2019 financial results and provide a corporate update on Thursday, November 7, 2019 (Press release, Audentes Therapeutics, OCT 31, 2019, View Source [SID1234550133]).

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The conference call will be held at 4:30 pm ET. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investor + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 8575077 to access the call.

A replay of the live webcast will be available on the Audentes website for approximately 30 days.

BioLife Solutions to Report Third Quarter 2019 Financial Results and Provide Business Update on November 12, 2019

On October 31, 2019 BioLife Solutions, Inc. (NASDAQ: BLFS), a leading developer and supplier of best-in-class bioproduction tools for cell and gene therapies, ("BioLife" or the "Company"), reported that the Company’s third quarter 2019 financial results will be released after market close on Tuesday, November 12, 2019, and that the Company will host a conference call and live webcast at 4:30 p.m. ET (1:30 p.m. PT) that afternoon (Press release, BioLife Solutions, OCT 31, 2019, View Source [SID1234550149]). Management will provide an overview of the Company’s financial results and a general business update.

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To access the webcast, log on to the Investor Relations page of the BioLife Solutions website at www.biolifesolutions.com/earnings. Alternatively, you may access the live conference call by dialing (844) 825-0512 (U.S. & Canada) or (315) 625-6880 (International) with the following Conference ID: 7207519. A webcast replay will be available approximately two hours after the call and will be archived on www.biolifesolutions.com for 90 days.

Autolus Therapeutics to Report Third Quarter 2019 Financial Results and Host Conference Call on November 7

On October 31, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that it will release its third quarter 2019 financial results and operational highlights before open of U.S. markets on Thursday, November 7, 2019 (Press release, Autolus, OCT 31, 2019, View Source [SID1234550684]).

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Management will host a conference call and webcast at 8:30 a.m. ET/1:30 p.m. GMT the same day to discuss the company’s financial results and provide a general business update. To listen to the webcast and view the accompanying slide presentation, please go to: View Source

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 5075598. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 5075598.

Three UCLA scientists receive grants totaling more than $18 million

On October 31, 2019 Three researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA reported that have received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the state’s stem cell agency (Press release, University of California at Los Angeles, OCT 31, 2019, View Source [SID1234553697]).

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The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute; Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Deng’s four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a person’s eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with the UCLA–UCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDA’s investigational new drug application process, which is required by the agency before a phase 1 clinical trial — the stage of testing that focuses on a treatment’s safety.

Chen’s two-year, $3.2 million award will fund efforts to create a more effective CAR T cell therapy for multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuo’s 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies — bubble baby disease and X-linked chronic granulomatous disease — with a similar therapy.