Bellicum Pharmaceuticals Announces Presentation on CAR-NK Cell Program at SITC 2019

On October 31, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported that an abstract for a preclinical investigation from its natural killer cell chimeric antigen receptor (CAR) program has been accepted for poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (Press release, Bellicum Pharmaceuticals, OCT 31, 2019, View Source [SID1234550118]). The meeting is being held November 6-10, 2019 in National Harbor, Md.

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Details of the poster presentation are as follows:

Title: Solid Tumor Cytotoxicity by Natural Killer Cells Expressing a HER2-Directed Chimeric Antigen Receptor Enhanced by MyD88/CD40 (MC)
Poster Board: #P151
Presenter: Henri Bayle, Ph.D.
Time/Location: Friday, November 8, 2019, 7 a.m. to 8 p.m. ET

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.

Aclaris Therapeutics to Announce Third Quarter 2019 Financial Results on November 7, 2019

On October 31, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS) a physician-led biopharmaceutical company focused on immuno-inflammatory diseases, reported it will report financial results for the third quarter 2019, Thursday, November 7th, after U.S. financial markets close (Press release, Aclaris Therapeutics, OCT 31, 2019, View Source [SID1234550116]).

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Management will conduct a conference call at 5:00 PM ET to discuss Aclaris’ financial results and provide a general business update. The conference call will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 2698128 prior to the start of the call.

Replimune to Host Investor Event at the 34th Annual Meeting of the Society for Immunotherapy of Cancer (SITC)

On October 31, 2019 Replimune Group Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported it will host an investor event to review the full safety, clinical efficacy, and biomarker data from Part 1 of the Phase 1/2 clinical trial of RP1 alone and in combination with Opdivo (Press release, Replimune, OCT 31, 2019, View Source [SID1234550115]). The event will include a presentation by Robert Coffin, Ph.D., President and CEO of Replimune who will be joined by the principle investigator, Mark Middleton, MD, PhD, Professor of Experimental Cancer at the University of Oxford, and other senior members of the Replimune management team. The presentation will focus on the data being presented at SITC (Free SITC Whitepaper) as well as additional supporting data from the clinical trial.

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The presentation will begin at 6:30 p.m. Eastern Time on Friday, November 8, 2019 in National Harbor, MD.

A simultaneous webcast will be available in the Investors section of Replimune’s website at www.replimune.com. A replay will be available for 30 days following the conference.

OB-002 Significantly Reduces Bone Metastasis in a Murine Model of Breast Cancer

On October 31, 2019 Orion Biotechnology Canada, Ltd. reported that OB-002 was able to significantly reduce bone metastasis in a murine model of breast cancer (Press release, Orion Biotechnology, OCT 31, 2019, View Source [SID1234550114]). The study was conducted in collaboration with ProQinase (Heidelberg, Germany; View Source). Female BALB/c mice received orthotopic implantation of 4T1-M3_Luc cells into the mammary fat pad. The 4T1 breast cancer cells were transfected with firefly luciferase that allowed bioluminescent quantification of metastatic disease. Mice were randomized to receive placebo, OB-002, or a combination of OB-002 and an anti-PD-1. The OB-002 was administered via intraperitoneal (IP) injection daily (Monday through Friday) and the anti-PD-1 was administered IP every 3-4 days. The mice randomized to receive OB-002 at a dose of 20 mg/kg demonstrated a significant reduction in Day 20 primary tumor volume compared to placebo and the mice randomized to receive 80 mg/kg had a significant reduction in spinal bone metastasis.

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"These data demonstrating that OB-002 can induce a significant reduction in both primary and metastatic breast cancer are very exciting and add to the growing body of data suggesting that OB-002 has the potential to play an important role in cancer therapy," said Dr. Ian McGowan, Chief Medical Officer for Orion Biotechnology. "Unfortunately, bone is the most common site of metastatic breast cancer (Ording AG et al., Clin Exp Metastasis 2017) and approximately 70% of women dying from breast cancer have bone metastases (Awolaran O et al., Breast 2016). Clearly, any product that has the potential to reduce breast cancer-associated bone metastasis would be a major advance in the field."

Mark Groper, President and CEO of Orion Biotechnology, added, "Breast cancer is the most common cancer in women worldwide and remains a challenging disease to manage. Bone metastasis is a frequent and debilitating complication of breast cancer and so these preliminary pre-clinical data are very encouraging."