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Syros Reports Fourth Quarter and Full Year 2018 Financial Results and Highlights Multiple Upcoming Clinical Milestones for Its First-in-Class Programs

On March 7, 2019 Syros Pharmaceuticals (NASDAQ: SYRS), a leader in the development of medicines that control the expression of genes, reported financial results for the fourth quarter and full year ended December 31, 2018 and provided an update on recent accomplishments and upcoming events (Press release, Syros Pharmaceuticals, MAR 7, 2019, View Source [SID1234534082]).

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"Our accomplishments in 2018 position us for multiple potential clinical milestones in 2019 and 2020 that bring us closer to our vision of building a fully integrated company with medicines that provide a profound benefit for patients," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "Building on promising clinical data for both our lead programs, we plan to expand our ongoing Phase 2 trial of SY-1425 in combination with azacitidine with the addition of a cohort in RARA and IRF8 biomarker-positive relapsed or refractory AML patients. We have also added a cohort of recurrent ovarian clear cell cancer patients to our ongoing Phase 1 trial of SY-1365. The unmet need in both these patient populations is significant, and we believe these cohorts could lead to rapid clinical proof-of-concept. These new cohorts, when combined with our ongoing evaluation of SY-1365 as a single agent in patients with high-grade serous ovarian cancer, give us three potential fast-to-market opportunities for our lead programs. We are focused on executing on our clinical trials with the aim of delivering much-needed therapies to patients as quickly as possible."

Upcoming Milestones:

SY-1425

Syros announced today that it plans to expand its ongoing Phase 2 trial to assess the safety and efficacy of SY-1425 in combination with azacitidine in RARA or IRF8 biomarker-positive patients with relapsed or refractory acute myeloid leukemia (AML). The Company expects this expansion to be open for enrollment in the third quarter.
Syros plans to complete enrollment in mid-2019 in the ongoing Phase 2 trial cohort evaluating the safety and efficacy of SY-1425 in combination with azacitidine in RARA or IRF8 biomarker-positive patients with newly diagnosed AML who are not suitable candidates for standard chemotherapy.
Syros plans to report updated data in the second half of 2019 from the ongoing Phase 2 trial of SY-1425 in combination with azaciditine in newly diagnosed unfit AML patients.
SY-1365

Syros announced today that it has added an expansion cohort to its ongoing Phase 1 trial of SY-1365 to evaluate its safety and efficacy as a single agent in patients with recurrent ovarian clear cell cancer. The Company expects the cohort to be open for enrollment in the second quarter of 2019. This cohort will replace the cohort evaluating SY-1365 in patients with primary platinum refractory ovarian cancer.
Syros plans to report initial clinical data in the fourth quarter of 2019 from the expansion portion of its ongoing Phase 1 trial. These data are expected to include initial efficacy and safety assessments from the cohort evaluating SY-1365 as a single agent in high-grade serous ovarian cancer patients who have had three or more prior lines of therapy; initial safety and pharmacokinetic data from the cohort evaluating SY-1365 in combination with carboplatin in high-grade serous ovarian cancer patients who have had one or more prior lines of therapy; and initial safety, efficacy and mechanistic data from the cohort evaluating SY-1365 as a single agent in patients with advanced solid tumors accessible for biopsy.
SY-5609

Syros plans to complete investigational new drug-enabling studies of SY-5609 in 2019 to support the initiation of a Phase 1 oncology trial in early 2020.
Recent Pipeline Highlights:

In December 2018, Syros’ collaborators presented new preclinical data on SY-1365 at the San Antonio Breast Cancer Symposium (SABCS). The data showed that SY-1365 inhibits tumor cell growth in hormone receptor-positive (HR-positive) breast cancer cell lines that are resistant to treatment with CDK4/6 inhibitors and that SY-1365 has synergistic activity in combination with fulvestrant in these treatment-resistant cells.
In December 2018, Syros presented initial clinical data from cohorts in its ongoing Phase 2 trial evaluating SY-1425 in combination with azacitidine at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. SY-1425 in combination with azacitidine showed high response rates and rapid onset of action in biomarker-positive patients with newly diagnosed unfit AML as of an October 29, 2018 data cut-off. The data showed:
The aggregate complete response (CR) and complete response with incomplete blood count recovery (CRi) rate was 50% in biomarker-positive patients (n=8), and the overall response rate (ORR) was 63%.
Most initial responses in biomarker-positive patients were seen at the end of the first treatment cycle, and duration of responses ranged from 29 to 337 days, with four of five responding patients remaining on treatment.
The ORR was 17% in biomarker-negative patients (n=6). While the data from this cohort were less mature due to the timing of patient enrollment, Syros believes that the difference in the observed ORR supports the potential predictive value of the RARA and IRF8 biomarkers for identifying patients most likely to respond to SY-1425.
The combination was generally well-tolerated, with no evidence of increased toxicities beyond what has been seen with either agent alone, including myelosuppression, which can occur when combining drugs to treat AML.
In December 2018, Syros also presented initial clinical data from a Phase 2 study cohort evaluating SY-1425 in combination with daratumumab in biomarker-positive patients with relapsed or refractory AML and higher-risk myelodysplastic syndrome at ASH (Free ASH Whitepaper). While the data showed that SY-1425 induced CD38 expression in eight of nine evaluable patients, CD38 expression increased to levels exceeding those of a multiple myeloma cell line control in only two of these patients. Of those two, one had a morphological leukemia-free state (MLFS) response. Based on these data, Syros made a portfolio prioritization decision not to pursue further development of SY-1425 in combination with daratumumab.
In November 2018, Syros presented clinical data from the dose escalation portion of its Phase 1 trial of SY-1365 in patients with advanced solid tumors at the 30th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium (EORTC-NCI-AACR) (Free EORTC-NCI-AACR Whitepaper). The data showed proof-of-mechanism and early signs of clinical activity at tolerable doses:
Clinical activity was observed in seven of 19 response-evaluable patients, including one patient with recurrent ovarian clear cell cancer who had a confirmed partial response (PR) and six additional patients who had stable disease (SD).
Dose-dependent effects on CDK7 occupancy and downstream gene expression changes in blood cells were observed.
At doses of 32 mg/m2 and higher, CDK7 occupancy was greater than 50% when measured three days following dose administration, exceeding target occupancy levels in preclinical models that correlated with anti-tumor activity.
Adverse events were predominately low-grade, reversible and manageable.
Also at EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper), Syros presented preclinical data on SY-1365 showing synergistic anti-tumor activity in combination with carboplatin in ovarian cancer cell lines and decreased expression of DNA damage response genes, providing a mechanistic rationale for the ongoing investigation of SY-1365 in combination with carboplatin in ovarian cancer patients.
Additionally, Syros presented the first preclinical data from its oral CDK7 program, detailing the selectivity, potency and anti-tumor activity of several compounds and supporting the selection of SY-5609, an oral CDK7 inhibitor, as a development candidate.
Recent Corporate Highlights:

In December 2018, Syros announced that it had been added to the NASDAQ Biotechnology Index (NASDAQ: NBI), effective Monday, December 24, 2018.
Fourth Quarter 2018 Financial Results

Cash, cash equivalents and marketable securities as of December 31, 2018 were $99.7 million, compared with $72.0 million on December 31, 2017.

For the fourth quarter of 2018, Syros reported a net loss of $18.0 million, or $0.54 per share, compared to a net loss of $15.3 million, or $0.58 per share, for the same period in 2017.

Revenues were $0.9 million for the fourth quarter of 2018, which relate entirely to Syros’ target discovery collaboration with Incyte Corporation. Syros did not record revenues in the fourth quarter of 2017.
Research and development (R&D) expenses were $15.1 million for the fourth quarter of 2018, as compared to $11.8 million for the same period in 2017. This increase was primarily attributable to an increase in SY-1365 contract manufacturing costs and professional fees in support of Syros’ clinical trials, as well as an increase in employee-related expenses.
General and administrative (G&A) expenses were $4.4 million for the fourth quarter of 2018, as compared to $3.7 million for the same period in 2017. This increase was primarily attributable to an increase in employee-related expenses.
Full Year 2018 Financial Results

For the full year ended December 31, 2018, Syros reported a net loss of $62.3 million, or $1.91 per share, compared to a net loss of $54.0 million, or $2.13 per share, for the same period in 2017.

Revenues for the year ended December 31, 2018 were $2.1 million, as compared to $1.1 million for the same period in 2017. Revenues earned in 2018 related entirely to the Incyte collaboration. Revenues earned in 2017 were earned from a research agreement with a multinational pharmaceutical company.
R&D expenses for the year ended December 31, 2018 were $50.2 million, as compared to $41.9 million for the same period in 2017. This increase was primarily attributable to an increase in contract manufacturing costs and professional fees in support of Syros’ clinical trials, as well as an increase in employee-related expenses.
G&A expenses for the year ended December 31, 2018 were $16.2 million, as compared to $13.9 million for the same period in 2017. This increase was primarily attributable to an increase in employee-related expenses.
Financial Guidance

Based on its current plans, Syros believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund its planned operating expenses and capital expenditure requirements into the second quarter of 2020.

Conference Call and Webcast:

Syros will host a conference call today at 8:30 a.m. ET to discuss these fourth quarter and full year 2018 financial results and provide a corporate update.

The live call may be accessed by dialing (866) 595-4538 for domestic callers or (636) 812-6496 for international callers and referencing conference ID number: 7258556. A live webcast of the conference call will be available online on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 90 days.

Rheos Medicines to Present at the Cowen and Company 39th Annual Health Care Conference

On March 7, 2019 Rheos Medicines, a biopharmaceutical company harnessing insights in immunometabolism to create a new class of therapeutics for patients with severe autoimmune disorders, inflammatory diseases and cancer, reported that Gad Soffer, the Company’s Chief Operating Officer, will present at the Cowen and Company 39th Annual Health Care Conference on Wednesday, March 13, 2019 at 3:30 p.m. EDT (Press release, Rheos Medicines, MAR 7, 2019, View Source [SID1234534081]). The Conference will be held at the Boston Marriott Copley Place in Boston, MA.

IMV Inc. to Present at the Cowen and Company 39th Annual Health Care Conference

On March 7, 2019 IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical stage immuno-oncology company reported that Pierre Labbé, IMV’s Chief Financial Officer will present at the Cowen and Company 39th Annual Health Care Conference, which will be held March 11-13, 2019 in Boston, MA (Press release, IMV, MAR 7, 2019, View Source [SID1234534080]).

IMV’s presentation details include:

Date: Wednesday, March 13, 2019
Time: 9:20 – 9:50 a.m. ET
Location: Salon C, 4th Floor at The Marriott Copley Hotel, Boston, MA
There will be a live webcast of IMV’s presentation accessible thru the IMV website at: http://wsw.com/webcast/cowen52/imv/ and the presentation will then be archived for 90 days afterwards. A copy of the presentation will also be available in the Events & Presentations section of IMV’s website: www.imv-inc.com.

Rocket Pharmaceuticals Reports Full Year 2018 Financial Results and Operational Highlights

On March 7, 2019 Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the year ended December 31, 2018, and provides an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAR 7, 2019, View Source [SID1234534079]).

"2018 was a great year for Rocket," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We successfully established ourselves as a public gene therapy company, funded our pipeline with more than $250 million in capital, achieved clinical proof of concept for our lead lentiviral gene therapy program in FA, expanded our pipeline with a new AAV gene therapy program in Danon disease, and received FDA clearance of three Rocket-sponsored INDs in only three months. These accomplishments are a testament to our ability to execute on our goals and commitment to bring new therapies to patients who desperately need them."

Dr. Shah added, "Looking ahead, Rocket is entering an exciting period in our evolution as we anticipate treating more patients with our gene therapy products across our pipeline. We look forward to at least two clinical readouts this year that, if successful, may support advancing our gene therapies that have the potential to transform the treatment paradigm in diseases that are limited to toxic bone marrow or organ transplant regimens. We believe we are well-positioned and prepared to meet all of these exciting challenges and opportunities."

Full Year 2018 and Recent Highlights

Expanded Pipeline with First AAV Gene Therapy Program Targeting a Monogenic Heart Failure Syndrome. In November 2018, the Company presented preclinical proof of concept data from its first adeno-associated viral vector (AAV)-based gene therapy, RP-A501, targeting Danon disease. RP-A501 is being developed in collaboration with Dr. Eric Adler, Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health and Professor of Medicine at University of California San Diego School of Medicine. Preclinical studies show RP-A501 improves patient survival and corrects the disease phenotype with dose-dependent improvements in molecular, structural, and functional endpoints with a clean safety and tolerability profile. The Company simultaneously announced an exclusive, worldwide license agreement with REGENXBIO Inc. for Rocket to develop and commercialize gene therapy treatments for Danon disease using REGENXBIO’s NAV AAV9 vector, as well as exclusive options for two additional NAV AAV vectors for the treatment of Danon disease. A Phase 1 clinical trial of RP-A501 is planned to commence in the second quarter of 2019.
Initial Clinical Proof of Concept Achieved in FA. Long-term clinical data from the ongoing Phase 1/2 clinical trial of RP-L102 for Fanconi Anemia (FA) utilizing "Process A", the first-generation non-optimized process, were presented at ESGCT in November 2018. Results showed durable engraftment at 30 months post treatment, stabilization of previously-declining blood counts and progressive increases in corrected versus non-corrected peripheral blood leukocytes. Moving forward, the Company plans to initiate a Phase 1 clinical trial of RP-L102 at Stanford University utilizing "Process B" which incorporates higher cell doses, transduction enhancers, and commercial-grade vector manufacturing and cell processing.
Three Gene Therapy INDs Cleared by the FDA. In a three-month period, the FDA cleared three Rocket-sponsored Investigational New Drug (IND) applications, paving the way for Phase 1 clinical trials to begin in FA and Danon disease along with a Phase 1/2 adaptive clinical study to begin in Leukocyte Adhesion Deficiency-I (LAD-I).
Nine Regulatory Designations Across Pipeline – RMAT, Fast Track, ATMP, Orphan Drug and Rare Pediatric Disease.
The Company received notification that the following programs received regulatory designations:
RP-L102 for FA received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Rare Pediatric Disease designations from the FDA, as well as Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA).
RP-L201 for LAD-I received Fast Track and Rare Pediatric Disease designations from the FDA, as well as ATMP classification from the EMA.
RP-L401 for Infantile Malignant Osteopetrosis received Orphan Drug designation from the FDA.
RP-A501 for Danon disease received Orphan Drug designation from the FDA.
Each designation provides numerous incentives to support the development of Rocket’s programs, including prescription drug user fee waivers, increased access to the regulatory authorities, expedited review timelines and tax credits towards the cost of clinical trials.
Approximately $150 Million Secured in Two Oversubscribed Public Offerings. Following the successful completion of its reverse merger with Inotek Pharmaceuticals in January 2018 that capitalized the Company with $97.6 million in cash, Rocket completed two oversubscribed underwritten public offerings of its common stock for gross proceeds of approximately $150 million over the course of the year.
Anticipated Milestones

FA (RP-L102)
Initiation of Phase 1 clinical trial of RP-L102 utilizing "Process B" (2Q19)
Additional data from RP-L102 utilizing "Process A" (2H19)
Initial data from Phase 1 clinical trial of RP-L102 utilizing "Process B" (2H19)
Regulatory alignment on final endpoints for registration (2H19)
LAD-I (RP-L201)
Initiation of Phase 1/2 clinical trial of RP-L201 in support of registration (2Q19)
Initial data from Phase 1 portion of clinical trial of RP-L201 (2H19)
PKD (RP-L301)
Initiation of Phase 1 clinical trial of RP-L301 (2H19)
Danon (RP-A501)
Initiation of Phase 1 clinical trial of RP-A501 (2Q19)
Upcoming Investor Conferences

Cowen and Company 39th Annual Health Care Conference. Rocket is scheduled to participate in a fireside chat on Tuesday, March 12, 2019, at 10:40 a.m. Eastern Time.
Alliance for Regenerative Medicine Cell & Gene Investor Day. Rocket is scheduled to present on Thursday, March 21, 2019, at 9:55 a.m. Eastern Time.
Fourth Quarter and Full Year 2018 Financial Results

Cash position. Cash, cash equivalents and investments as of December 31, 2018, were $213.1 million.
Debt. Our cash position includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $23.7 million and $53.3 million for the three and twelve months ended December 31, 2018, compared to $4.9 million and $14.9 million for the three and twelve months ended December 30, 2017.
G&A expenses. General and administrative expenses were $2.9 million and $17.9 million for the three and twelve months ended December 31, 2018, compared to $1.7 million and $4.9 million for the three and twelve months ended December 31, 2017.
Net loss. Net loss was $27.3 million and $74.5 million or $(0.66) and $(1.89) per share (basic and diluted) for the three and twelve months ended December 31, 2018, compared to $6.7 million and $19.6 million or $(0.98) and $(2.88) per share (basic and diluted) for the three and twelve months ended December 31, 2017.
Shares outstanding. Approximately 45.2 million shares of common stock were outstanding as of December 31, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of December 31, 2018, will be sufficient to run its operations into the second half of 2020.

ATARA BIOTHERAPEUTICS TO PARTICIPATE IN UPCOMING CONFERENCES

On March 7, 2019 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported that members of the Company’s management team will participate at several upcoming conferences (Press release, Atara Biotherapeutics, MAR 7, 2019, View Source [SID1234534078]):

Cowen and Company 39th Annual Health Care Conference

Isaac Ciechanover, M.D., the Company’s President and Chief Executive Officer, will give a presentation on Tuesday, March 12, 2019 at 10:00 a.m. EDT. The conference will be held at the Boston Marriott Copley Place in Boston, MA.
Memorial Sloan Kettering 3rd Annual Clinical Application of CAR T Cells Conference

Chris Haqq, M.D., Ph.D., the Company’s Executive Vice President of Research and Development and Chief Scientific Officer, will participate in the Industry Session on Thursday, March 14, 2019 at 4:35 p.m. EDT. The conference will be held at the Zuckerman Research Center in New York, NY.
William Blair 9th Annual Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper)

Chris Haqq, M.D., Ph.D., the Company’s Executive Vice President of Research and Development and Chief Scientific Officer, will present on Wednesday, March 20, 2019 at 11:00 a.m. EDT. The conference will be held at the Apella Event Space Alexandria Center in New York, NY.
Alliance for Regenerative Medicine 7th Annual Cell & Gene Therapy Investor Day

Chris Haqq, M.D., Ph.D., the Company’s Executive Vice President of Research and Development and Chief Scientific Officer, will participate in a panel titled "Cell Therapy for Solid Tumors" on Thursday, March 21, 2019 at 1:30 p.m. EDT. The Conference will be held at the Metropolitan Club in New York, NY.
Jefferies 6th Annual IO Cell Therapy Summit

Dietmar Berger, M.D., Ph.D., the Company’s Global Head of Research and Development, will present on Friday, April 5, 2019. The conference will be held at the Boston Harbor Hotel in Boston, MA and the time of the presentation will be available on Atara’s investor events webpage by March 29.
Live audio webcasts of the presentations at the Cowen and Company 39th Annual Health Care Conference and Alliance for Regenerative Medicine 7th Annual Cell & Gene Therapy Investor Day will be available by visiting the Investor Events and Presentations section of the Atara website. An archived replay of each webcast will be available on the Company’s website for 14 days following the conclusion of each presentation.