On October 31, 2019 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the third quarter ended September 30, 2019 (Press release, bluebird bio, OCT 31, 2019, View Source [SID1234550119]).

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"During the third quarter we advanced our country-by-country launch plans in Europe and, with the recent approval of the commercial drug product manufacturing specifications for ZYNTEGLO, we moved one step closer to our goal of treating patients suffering from TDT in early 2020," said Nick Leschly, chief bluebird. "Also this quarter, we presented updated data from the Phase 2/3 Starbeam study in patients with CALD. To report that patients continued to be free of MFDs at up to five years of follow-up is something we’re tremendously proud to do for these families, and we look forward to advancing that program in the regulatory process next year. Looking ahead, we plan to provide clinical updates for ZYNTEGLO and across the rest of our portfolio, including LentiGlobin in sickle cell disease, bb21217 in multiple myeloma, and from our registration-enabling KarMMa study of ide-cel in patients with multiple myeloma by the end of this year. I’d like to thank all the bluebirds around the globe for their tireless focus on doing the right thing for our patients – we’ve seen amazing progress thus far in 2019 and I look forward to ending the year on a strong note."

Recent Highlights:

TDT

ZYNTEGLO COMMERCIAL READINESS – In October, bluebird bio announced that the European Medicines Agency (EMA) approved the refined commercial drug product manufacturing specifications for ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene), a one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. With this update, apceth is in the final stages of preparing to manufacture ZYNTEGLO for commercial use. The company continues to proceed with discussions on value-based payment agreements and Qualified Treatment Center contracts and expects to treat the first commercial patient in early 2020.
CALD

DATA FROM STARBEAM STUDY (ALD-102) AND ALD-103 PRESENTED – At the 13thEuropean Pediatric Neurology Society (EPNS) Congress in September 2019, bluebird bio presented new data from the clinical development program for its investigational studies of Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy: updated data from the Phase 2/3 Starbeam study (ALD-102) in boys 17 years of age and under with CALD and updated data from the ongoing observational study (ALD-103) of allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys 17 years of age and under with CALD. Long-term follow-up data as of April 2019 showed that the 88% of patients treated in the Starbeam study (ALD-102) were free of major functional disabilities (MFDs) at two years, and continued to remain MFD-free at up to five years of follow-up.
COMPANY

FIRST PATIENT TREATED IN PHASE 1/2 TRIAL FOR MERKEL CELL CARCINOMA (MCC) – In August 2019, Fred Hutchinson Cancer Research Center infused the first patient in their proof-of-concept phase 1/2 single-arm study evaluating Merkel Cell Polyomavirus (MCPyV) TCR-engineered autologous T cells in combination with avelumab (anti-PDL1) for the treatment of MCC. Results from the academic phase 1/2 single-arm study are expected to inform next-generation T cell approaches including TCR engineering and checkpoint inhibition. The study will enroll approximately 16 patients. Development of this program is led by Fred Hutchinson Cancer Research Center. bluebird bio retains the exclusive option to license this program.
NOVO NORDISK COLLABORATION – In October 2019, bluebird bio and Novo Nordisk announced a research collaboration to jointly develop next-generation in vivo genome editing treatments for genetic diseases, including hemophilia. During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development gene therapy candidate with the ambition of offering people with hemophilia A a lifetime free of factor replacement therapy.
MANAGEMENT UPDATE – In October 2019, bluebird bio announced that Jeffrey T. Walsh, chief strategy officer, has decided to transition from his current role effective January 6, 2020. Jeff has not only built a strong foundation for bluebird’s overall growth strategy but also leaves an experienced and passionate team. Both Chip Baird, chief financial officer, and Joanne Smith-Farrell, chief business officer, will assume broader corporate development and strategic responsibilities as bluebird continues to deliver on its mission for patients.
NEW BOARD APPOINTMENT – In September 2019, bluebird bio announced the appointment of William R. Sellers, M.D. to its Board of Directors.
Upcoming Anticipated Milestones:

TDT
Initiation of a rolling Biologics Licensing Application submission to the U.S. FDA for ZYNTEGLO in patients with TDT and non-β0/β0 genotypes by the end of 2019
Presentation of ZYNTEGLO clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non-β0/β0 genotypes by the end of 2019
Presentation of ZYNTEGLO clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and a β0/β0 genotype or an IVS-I-110 mutation by the end of 2019
SCD
Phase 3 HGB-210 study of LentiGlobin in patients with SCD open and enrolling by the end of 2019
Presentation of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD by the end of 2019
Multiple Myeloma
Ide-cel clinical data update from the registration-enabling KarMMa study in patients with relapsed/refractory multiple myeloma by the end of 2019
Presentation of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma by the end of 2019
Third Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2019 and December 31, 2018 were $1.41 billion and $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities and cash used to purchase property, plant and equipment, including those purchases related to the company’s buildout of its manufacturing facility in Durham, North Carolina.
Revenues: Collaboration and license and royalty revenues were $8.9 million for the three months ended September 30, 2019 compared to $11.5 million for the three months ended September 30, 2018. Collaboration and license and royalty revenues were $34.7 million for the nine months ended September 30, 2019 compared to $35.3 million for the nine months ended September 30, 2018. The decrease in both periods was primarily attributable to a decrease in collaboration revenue under our arrangement with Celgene, partially offset by an increase in license and royalty revenue and collaboration revenue under our arrangement with Regeneron.
R&D Expenses: Research and development expenses were $151.4 million for the three months ended September 30, 2019 compared to $116.7 million for the three months ended September 30, 2018. Research and development expenses were $420.6 million for the nine months ended September 30, 2019 compared to $328.9 million for the nine months ended September 30, 2018. The increase in both periods was primarily driven by costs incurred to advance and expand the company’s pipeline.
G&A Expenses: General and administrative expenses were $66.3 million for the three months ended September 30, 2019 compared to $44.5 million for the three months ended September 30, 2018. General and administrative expenses were $195.2 million for the nine months ended September 30, 2019 compared to $120.6 million for the nine months ended September 30, 2018. The increase in both periods was largely attributable to costs incurred to support the company’s ongoing operations and growth of its pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $206.0 million for the three months ended September 30, 2019 compared to $145.5 million for the three months ended September 30, 2018. Net loss was $566.3 million for the nine months ended September 30, 2019 compared to $406.6 million for the nine months ended September 30, 2018.

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

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.

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.

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.