Cytokinetics, Inc. Reports Third Quarter 2017 Financial Results

On October 26, 2017 Cytokinetics, Incorporated (Nasdaq:CYTK) reported total revenues for the third quarter of 2017 were $6.2 million, compared to $59.0 million, during the same period in 2016. Net loss for the third quarter was $32.4 million, or $0.60 per basic and diluted share, respectively, compared to net income for the same period in 2016 of $33.4 million, or $0.84 and $0.77 per basic and diluted share, respectively (Press release, Cytokinetics, OCT 26, 2017, View Source [SID1234521210]). As of September 30, 2017, cash, cash equivalents and investments totaled $308.2 million.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

“We are proud of the key milestones we recently achieved in preparation for the planned release of results from VITALITY-ALS, and potential regulatory filings and commercialization of tirasemtiv in North America and Europe,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “In the third quarter, we also advanced CK-2127107 into two additional mid-stage clinical trials, one in patients with ALS and another in elderly subjects with limited mobility; we look forward to data from four clinical trials in this program in 2018. Finally, we are pleased that patient enrollment in GALACTIC-HF continues on target and commenced in Japan triggering a $10 million milestone payment to Cytokinetics.”

Recent Highlights and Upcoming Milestones

Skeletal Muscle Program

tirasemtiv (fast skeletal muscle troponin activator)

Completed dosing of patients in VITALITY-ALS (Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices after Treatment for a Year in ALS), our Phase 3 clinical trial of tirasemtiv in patients with ALS.

Continued enrollment in VIGOR-ALS (Ventilatory Investigations in Global Open-Label Research in ALS), an open-label clinical trial designed to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who have completed participation in VITALITY-ALS.

Conducted clinical, regulatory, non-clinical and other activities intended to support potential regulatory filings and registration of tirasemtiv in North America and Europe.

Conducted manufacturing, logistical planning, market research, market access and other commercial readiness activities intended to support potential registration and commercialization of tirasemtiv in North America and Europe.

Proceeding to collection of final data from VITALITY-ALS and clinical trial database lock in Q4 2017. We plan to conduct analyses of data from VITALITY-ALS and present results from this clinical trial on December 8, 2017 at the 28th International Symposium on ALS/MND in Boston.

Expect to continue to enroll patients who complete VITALITY-ALS into VIGOR-ALS throughout 2017.

CK-2127107 (next-generation fast skeletal muscle troponin activator)

Announced the start of a Phase 1b, double-blind, randomized, placebo-controlled, multiple dose, two-period crossover study to assess the effect of CK-2127107 on measures of physical function in elderly adults with limited mobility. This study is being conducted by Astellas, in collaboration with Cytokinetics.
Announced the start of FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS). This Phase 2 clinical trial is designed to assess the change from baseline in the percent predicted slow vital capacity (SVC) and other measures of skeletal muscle function after 12 weeks of treatment with CK-2127107 in patients with ALS. This trial is being conducted by Cytokinetics, in collaboration with Astellas.
“Reasons for Screen Failures and Baseline Characteristics of Randomized Patients from the First Cohort of the Phase 2 Clinical Trial of CK-2127107 in Patients with SMA,” presented by Stacy Rudnicki, M.D., Director, Clinical Research, Cytokinetics, at the Cure SMA 2017 Annual SMA Conference in Orlando, FL.
Expect to complete enrollment of Cohort 2 of the Phase 2 clinical trial of CK-2127107 in patients with SMA in 2017.
Expect data from the Phase 2 clinical trial of CK-2127107 in patients with SMA in Q1 2018.
Expect Astellas to continue enrollment in a Phase 2 clinical trial of CK-2127107 in patients with COPD in 2017.
Expect Astellas to continue enrollment in a Phase 1b clinical trial of CK-2127107 in adults with limited mobility in 2017.

Expect to continue enrollment in FORTITUDE-ALS, a Phase 2 clinical trial of CK-2127107 in patients with ALS in 2017.
Cardiac Muscle Program

omecamtiv mecarbil (cardiac muscle myosin activator)

Announced that the Phase 2 clinical trial of omecamtiv mecarbil in Japanese patients with heart failure met its pharmacokinetic primary endpoint and demonstrated statistically significant improvements in systolic ejection time (SET), a secondary endpoint.

Announced that the first patient has been dosed in Japan 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 which is being conducted by Amgen, in collaboration with Cytokinetics. Coincident with patient dosing in Japan, Cytokinetics earned a $10 million milestone payment from Amgen.

Announced that additional results from COSMIC-HF (Chronic Oral Study of Myosin Activation to Increase Contractility in Heart Failure), a Phase 2 trial evaluating omecamtiv mecarbil in patients with chronic heart failure, were presented by John Teerlink, M.D., Professor of Clinical Medicine at the University of California San Francisco and Director of Heart Failure at the San Francisco Veterans Affairs Medical Centers in a Rapid Fire Abstracts Presentation at the 21st Annual Heart Failure Society of America Scientific Meeting in Dallas, TX. The results suggest that omecamtiv mecarbil may produce similar results with regard to cardiac function, heart rate, biomarkers and adverse events in patients with ischemic and non-ischemic heart failure due to left ventricular systolic dysfunction.

Continued to enroll patients in GALACTIC-HF, the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil, conducted by Amgen, in collaboration with Cytokinetics.

Expect continued enrollment of patients with chronic heart failure in GALACTIC-HF throughout 2017.
Pre-Clinical Research

Continued research activities under our joint research program with Amgen directed to the discovery of next-generation cardiac muscle activators and under our joint research program with Astellas directed to the discovery of next-generation skeletal muscle activators. In addition, company scientists continued independent research activities directed to our other muscle biology programs.
Expect to nominate at least 2 compounds from ongoing Research programs (partnered and unpartnered) as potential drug candidates by the end of 2017.
Financials

Revenues for the three and nine months ended September 30, 2017 were $6.2 million and $13.4 million, respectively, compared to $59.0 million and $73.3 million for the corresponding periods in 2016. Revenues for the first nine months of 2017 included the $10 million milestone payment from Amgen as well as $8.8 million of research and development revenues and $6.7 million of license revenues from our collaboration with Astellas and $1.3 million of research and development revenues from our collaboration with Amgen. Revenues for the first nine months of 2017 were offset by $13.8 million (out of the total of $40 million) for payments to Amgen related to our option to co-fund the Phase 3 development program of omecamtiv mecarbil in exchange for an increased royalty upon potential commercialization. Revenues in 2016 were primarily due to license revenue from the September 2016 expansion of our collaboration with Astellas. Astellas paid us $65 million in connection with the expanded collaboration.

Total research and development expenses for the three and nine months ended September 30, 2017 increased to $24.9 million and $64.0 million, respectively, from $17.9 million and $41.1 million for the same periods in 2016, primarily due to increased clinical activity, including activity for VITALITY-ALS and other activities intended to support potential regulatory filings and registration of tirasemtiv in North America and Europe, increased CK-2127107 clinical trials activity, as well as increased personnel.

General and administrative expenses for the three and nine months ended September 30, 2017 increased to $9.7 million and $26.2 million from $7.2 million and $21.1 million for the same periods in 2016, primarily due to increased personnel, non-cash stock compensation expense and commercial readiness activities.

Financial Guidance

The Company also announced updated financial guidance for 2017. The Company anticipates cash research and development expenses will be in the range of $103 to $107 million, cash revenue will be in the range of $16 to $18 million, and cash general and administrative expenses will remain in the range of $30 million to $32 million.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s third quarter 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 46689063.

An archived replay of the webcast will be available via Cytokinetics’ website until November 2, 2017. 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 46689063 from October 26, 2017 at 7:30 PM Eastern Time until November 2, 2017.

Exelixis to Present at the Credit Suisse 26th Annual Healthcare Conference on November 7th

On October 26, 2017 Exelixis, Inc. (NASDAQ: EXEL) reported that Michael M. Morrissey, Ph.D., the company’s President and Chief Executive Officer, will provide an overview of the company at the Credit Suisse 26th Annual Healthcare Conference taking place November 6-8 in Scottsdale, AZ (Press release, Exelixis, OCT 26, 2017, View Source;p=RssLanding&cat=news&id=2311868 [SID1234521212]). The Exelixis presentation is scheduled for 5:20 PM EST / 2:20 PM PST on Tuesday, November 7, 2017.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Know more, wherever you are:
Latest on Exelixis Cancer Pipeline, book your free 1stOncology demo here.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the presentation to ensure adequate time for any software download that may be required to listen to the webcast. A replay will also be available at the same location for 14 days.

Acceleron to Webcast Third Quarter 2017 Financial Results on November 7, 2017

On October 26, 2017 Acceleron Pharma Inc. (NASDAQ:XLRN) reported it will host a webcast and conference call on Tuesday, November 7, 2017 at 8:00 a.m. EST to discuss its third quarter 2017 financial results and provide an update on recent clinical development and corporate activities(Press release, Acceleron Pharma, OCT 26, 2017, View Source [SID1234521191]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The webcast will be accessible under “Events & Presentations” in the Investors/Media page of the company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the “Acceleron Third Quarter Earnings Call.”

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

Transgene: First Patient Treated in a Phase 1/2a Trial (Oncovirac) of Novel Oncolytic Virus TG6002 in Recurrent Glioblastoma

On October 26, 2017 Transgene (Paris:TNG) (Euronext Paris: TNG), a biotech company that designs and develops viral-based immunotherapies, reported that the first patient with recurrent glioblastoma has been treated at La Pitié-Salpêtrière hospital, Greater Paris University Hospitals, AP-HP (Paris), in the first-in-human clinical trial (Oncovirac trial) of TG6002, a novel oncolytic virus (Press release, Transgene, OCT 26, 2017, View Source [SID1234521218]). TG6002 represents the next generation of oncolytic virus (OV), which is administered intravenously and has multiple functions. It has been engineered to combine oncolysis (the breakdown of cancer cells) with the local production of 5-FU chemotherapy agent in the tumor. It is also expected to induce an immune response following the antigen spreading that is caused by the cancer cells’ breakdown.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

TG6002: a novel oncolytic virus allowing the targeted production of chemotherapy in the tumor

TG6002 is a next generation oncolytic immunotherapy, which has a double mechanism of action. It has been designed by Transgene to:

1. induce the breakdown of cancer cells (oncolysis) by tumor-selective viral replication. In preclinical experiments, TG6002 was able to induce response in the primary tumor and an immune-mediated regression of distant metastases (immunogenic cell death);

2. allow the local production of chemotherapy (5-FU), a widely used cancer chemotherapy, in the tumor. TG6002 expresses the proprietary Fcu1 gene in the cancer cells it has infected, leading to the local conversion of the 5-FC into 5-FU.

First-in-human trial to deliver first readouts in H2 2018

Oncovirac is an open-label Phase 1/2a trial evaluating the safety and tolerability of multiple-ascending doses of TG6002 administered intravenously in combination with oral 5-FC, a non-cytotoxic pro-drug, flucytosine, that can be converted in 5-FU. The anti-tumor activity of this novel oncolytic virus will also be monitored. The study will enroll patients suffering from recurrent glioblastoma, who have failed standard of care treatment.

Dr. Ahmed Idbaih, M.D., PhD, neuro-oncologist at La Pitié-Salpêtrière Hospital (Paris, France), is the principal investigator of the study. He is involved in several clinical trials dedicated to primary brain tumor patients. He also coordinates “GlioTex”, a research group focused on glioblastoma and experimental therapeutics at ICM (The Institut du Cerveau et de la Moelle épinière – Brain & Spine Institute). AP-HP Paris Greater Hospitals, is the sponsor of Oncovirac, a trial also supported by INCa (French National Cancer Institute). More information on the trial is available on clinicaltrials.gov (NCT03294486). The first readouts of the study are expected in the second half of 2018.

Maud Brandely, M.D., PhD, Chief Medical Officer of Transgene, added: “TG6002 is a very promising new generation of oncolytic virus, which has the potential to be administered intravenously. Based on our compelling preclinical data, we have established that its replication induces immunogenic cell lysis and the local production of chemotherapy. We are excited to see this novel immunotherapy with multiple modes of action enter the clinic and look forward to obtaining results that will allow further development of TG6002 in several solid tumors indications.”

Dr. Ahmed Idbaih, M.D., PhD, neuro-oncologist at La Pitié-Salpêtrière hospital, AP-HP, and principal investigator of the trial, added: “Current treatments of recurrent glioblastoma are insufficient. By combining the immunogenic lysis of cancer cells with the targeted production of chemotherapy in the tumor, TG6002 has the potential to show anti-tumor efficacy and to avoid systemic side effects of chemotherapy. We are very pleased to be conducting this first in human clinical trial evaluating this novel immunotherapy that we believe could improve the overall survival of recurrent glioblastoma patients while preserving their quality of life.”

About TG6002
TG6002 is a next generation oncolytic immunotherapy. It has been designed to induce the breakdown of cancer cells (oncolysis) and allow the local production of chemotherapy (5-FU) in the tumor. TG6002 is a modified Vaccinia virus, with double gene deletion (TK-RR-), and expressing the proprietary Fcu1 gene in the cancer cells it has infected, leading to the local conversion of the non-cytotoxic pro-drug, flucytosine (5-FC), into 5-FU, a widely used cancer chemotherapy. The oncolytic virus TG6002 has shown efficacy and good safety profile in several preclinical models of glioblastoma in vitro (i.e. cell line) and in vivo (i.e. xenografts in Swiss/Nude mice). Transgene believes that TG6002 may represent a new therapeutic option in recurrent glioblastoma patients. TG6002 could also be investigated in other solid tumors.

About Glioblastoma
Glioblastoma is the most common and the most aggressive primary brain cancer in adults. Approximately 70,000 new cases are diagnosed each year in Europe 28 and in the USA (Globocan 2012). Despite very intensive treatments (i.e. maximal safe surgery, radiotherapy, and several lines of cytotoxic chemotherapy), inducing significant adverse events, the prognosis of glioblastoma patients remains poor. More efficient and less toxic therapies are urgently needed to improve survival and quality of life of glioblastoma patients.

First Patient Treated in a Phase 1/2a Trial (Oncovirac) of Novel Oncolytic Virus TG6002 in Recurrent Glioblastoma

On October 26, 2017 Transgene (Euronext Paris: TNG), a biotech company that designs and develops viral-based immunotherapies, reported that the first patient with recurrent glioblastoma has been treated at La Pitié-Salpêtrière hospital, Greater Paris University Hospitals,AP-HP (Paris), in the first-in-human clinical trial (Oncovirac trial) of TG6002, a novel oncolytic virus (Press release, Transgene, OCT 26, 2017, View Source [SID1234521244]). TG6002 represents the next generation of oncolytic virus(OV), which is administered intravenously and has multiple functions. It has been engineered to combine oncolysis (the breakdown of cancer cells) with the local
production of 5-FU chemotherapy agent in the tumor. It is also expected to induce an immune response
following the antigen spreading that is caused by the cancer cells’ breakdown.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

TG6002: a novel oncolytic virus allowing the targeted production of chemotherapy in the tumor
TG6002 is a next generation oncolytic immunotherapy, which has a double mechanism of action. It has been
designed by Transgene to:
1. induce the breakdown of cancer cells (oncolysis) by tumor-selective viral replication. In preclinical
experiments, TG6002 was able to induce response in the primary tumor and an immune-mediated
regression of distant metastases (immunogenic cell death);
2. allow the local production of chemotherapy (5-FU), a widely used cancer chemotherapy, in the
tumor. TG6002 expresses the proprietary Fcu1 gene in the cancer cells it has infected, leading to
the local conversion of the 5-FC into 5-FU.

First-in-human trial to deliver first readouts in H2 2018
Oncovirac is an open-label Phase 1/2a trial evaluating the safety and tolerability of multiple-ascending doses
of TG6002 administered intravenously in combination with oral 5-FC, a non-cytotoxic pro-drug, flucytosine,
that can be converted in 5-FU. The anti-tumor activity of this novel oncolytic virus will also be monitored.
The study will enroll patients suffering from recurrent glioblastoma, who have failed standard of care
treatment.

Dr. Ahmed Idbaih, M.D., PhD, neuro-oncologist at La Pitié-Salpêtrière Hospital (Paris, France), is the principal
investigator of the study. He is involved in several clinical trials dedicated to primary brain tumor patients. He
also coordinates "GlioTex", a research group focused on glioblastoma and experimental therapeutics at ICM (The
Institut du Cerveau et de la Moelle épinière – Brain & Spine Institute). AP-HP Paris Greater Hospitals, is the
sponsor of Oncovirac, a trial also supported by INCa (French National Cancer Institute). More information on the
trial is available on clinicaltrials.gov (NCT03294486). The first readouts of the study are expected in the second
half of 2018.

Maud Brandely, M.D., PhD, Chief Medical Officer of Transgene, added: "TG6002 is a very promising new
generation of oncolytic virus, which has the potential to be administered intravenously. Based on our
compelling preclinical data, we have established that its replication induces immunogenic cell lysis and the
local production of chemotherapy. We are excited to see this novel immunotherapy with multiple modes of
action enter the clinic and look forward to obtaining results that will allow further development of TG6002
in several solid tumors indications."

Dr. Ahmed Idbaih, M.D., PhD, neuro-oncologist at La Pitié-Salpêtrière hospital, AP-HP, and principal
investigator of the trial, added: "Current treatments of recurrent glioblastoma are insufficient. By combining
the immunogenic lysis of cancer cells with the targeted production of chemotherapy in the tumor, TG6002
has the potential to show anti-tumor efficacy and to avoid systemic side effects of chemotherapy. We are
very pleased to be conducting this first in human clinical trial evaluating this novel immunotherapy that we
believe could improve the overall survival of recurrent glioblastoma patients while preserving their quality
of life."