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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]).

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"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.

ArQule Reports Fourth Quarter and Full Year 2018 Financial Results

On March 7, 2019 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the fourth quarter and full year of 2018 (Press release, ArQule, MAR 7, 2019, View Source [SID1234534077]).

For the quarter ended December 31, 2018, the Company reported a net loss of $8,487,000 or $0.08 per share, compared with net loss of $7,760,000 or $0.09 per share, for the quarter ended December 31, 2017. The Company reported a net loss of $15,482,000 or $0.16 per share, for the year ended December 31, 2018, compared with a net loss of $29,203,000 or $0.39 per share, for the year ended December 31, 2017.

As of December 31, 2018, the Company had a total of approximately $99,558,000 in cash, equivalents, and marketable securities.

Key Highlights from 2018

ARQ 531, our potent and reversible dual inhibitor of both wild-type and C481S-mutant BTK. Successfully progressedrecruitment in our ongoing phase 1 dose escalation trial in 2018, enrolling over 20 patients at 7 dose cohorts ranging from 5mg to 65mg QD. Data from this trial was presented at 3 major conferences (AACR, EHA (Free EHA Whitepaper), ASH (Free ASH Whitepaper)) in 2018 and demonstrated a good safety profile, profound target engagement and encouraging signs of dose-dependent clinical activity in both lymphomas and C481S-mutant CLL
Miransertib, our potent and selective first-generation AKT inhibitor. Presented first-of-its kind clinical data in Proteus syndrome and PROS at the American Society of Human Genetics (ASHG), received FDA Fast Track Designation in PROS, and worked with regulators to define registrational trial designs for both indications
ARQ 751, our highly potent and selective next-generation AKT inhibitor. Progressed the phase 1 basket trial in R/R or metastatic cancer patients harboring an AKT, PI3K or PTEN mutation, identified a recommended phase 2 dose of 75mg QD and presented data at the EORTC/AACR/NCI congress in November
Derazantinib, our FGFR inhibitor, partnered with Basilea and Sinovant, in a registrational trial for intrahepatic cholangiocarcinoma. Continued the timely recruitment and transfer of clinical and other responsibilities to Sinovant and Basilea following the outlicensing to both companies in February and April of last year, respectively
Capital Structure. Strengthened our capital structure through business development activities, which included funding from our collaborators and a successful offering of our common stock which raised gross proceeds of about $70 million
Stock Index Inclusions. Added to the family of Russell 2000 Index companies in June and the NASDAQ Biotechnology Index in December
Key Catalysts & Goals for 2019

ARQ 531: Dual BTK Inhibitor in B-Cell Malignancies

Complete the dose escalation portion of phase 1 trial
Determine a recommended phase 2 dose and initiate expansion cohort(s)
Present results of dose escalation phase 1 trial at major industry conference(s)
Miransertib: AKT Inhibitor in Proteus syndrome and PROS

Finalize regulatory interactions with the FDA
Initiate registrational trial cohorts in both Proteus syndrome and PROS
ARQ 751 (and Miransertib): AKT Inhibitors in Oncology

Complete recruitment in ongoing clinical trials
Present data sets at major medical conferences this year
Derazantinib: FGFR Inhibitor in iCCA

Complete the orderly and timely transfer of all clinical, manufacturing and regulatory responsibilities to our partners, Basilea and Sinovant
"2018 was a watershed year for ArQule," remarked Paolo Pucci, Chief Executive Officer. "The clinical progress we made across the entire portfolio positions us well for an even more transformational 2019. Specifically in rare disease, we now have the ability to expand the scope of the miransertib registrational trial beyond Proteus syndrome to the PROS family of overgrowth spectrum disorders which represents a particularly exciting opportunity because of their significantly larger prevalence."

Dr. Brian Schwartz, Chief Medical Officer, added "We made tremendous progress in 2018, enrolling over 20 patients across 7 cohorts in our dose escalation study with ARQ 531 which is now positioned as the first and best in class drug candidate to address the emerging medical need in BTK mutated malignancies. We are looking forward to advance ARQ 531, miransertib and ARQ 751 respectively into the next phase of development. I want to thank our collaborators such as the Ohio State University, the National Institute of Health and Bambino Gesu’, and others for their continued support in advancing these programs."

Revenues and Expenses

Revenues for the quarter ended December 31, 2018, were $2,941,000 compared with revenues of zero for the quarter ended December 31, 2017. Revenues for the year ended December 31, 2018 were $25,764,000 compared with revenues of zero for the year ended December 31, 2017. Research and development revenue increased in 2018 due to revenue of $5.9 million from our February 2018 Sinovant licensing agreement, $18.5 million from our April 2018 Basilea licensing agreement and $1.3 million from a non-exclusive license agreement for certain of our library compounds.

Research and development expenses in the fourth quarter of 2018 were $8,850,000 compared with $4,721,000 for the fourth quarter 2017. Fiscal 2018 research and development expenses were $28,710,000 compared with $19,468,000 for fiscal 2017. The $4.1 million increase in research and development expense in the fourth quarter of 2018 compared with the fourth quarter of 2017 was primarily due to higher outsourced preclinical, clinical and product development costs of $3.5 million and $0.6 million from labor and related costs.

The $9.2 million increase in research and development expense in 2018 was primarily due to higher outsourced preclinical, clinical and product development costs of $8.5 million and $0.7 million from labor and related costs.

General and administrative expenses in the fourth quarter of 2018 were $2,739,000, compared with $1,849,000 for the fourth quarter of 2017. General and administrative expenses for fiscal 2018 were $10,753,000, compared to $7,551,000 for fiscal 2017. The $0.9 million increase in general and administrative expense in the fourth quarter of 2018 compared with the fourth quarter of 2017 was principally due to higher consulting and professional fees of $0.4 million and labor and related costs of $0.5 million. The $3.2 million increase in general and administrative expense in 2018 was principally due to higher consulting and professional fees of $2.1 million and labor and related costs of $1.1 million.

2019 Financial Guidance

For 2019, ArQule expects revenue to range between $3 and $5 million. Net loss is expected to range between $40 and $43 million, and net loss per share to range between $(0.37) and $(0.39) for the year. ArQule expects to end 2019 with between $60 and $63 million in cash and marketable securities.

Conference Call and Webcast

ArQule will hold its fourth quarter and full year financial results call today, March 7, 2019 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events and Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. A replay will be available two hours after the completion of the call and can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events and Presentations."

Bristol-Myers Squibb to Announce Results for First Quarter 2019 on April 25, 2019

On March 7, 2019 Bristol-Myers Squibb Company (NYSE:BMY) reported that it will announce results for the first quarter of 2019 on Thursday, April 25, 2019 (Press release, Bristol-Myers Squibb, MAR 7, 2019, View Source [SID1234534073]). During a conference call at 10:30 a.m. ET on April 25, company executives will review financial information and will address inquiries from investors and analysts.

Investors and the general public are invited to listen to a live webcast of the call at View Source or by dialing in the U.S. toll free 888-254-3590 or international 720-543-0302, confirmation code: 7211894. Materials related to the call will be available at the same website prior to the conference call. A replay of the call will be available beginning at 1:45 p.m. ET on April 25 through 1:45 p.m. ET on May 9, 2019. The replay will also be available through View Source or by dialing in the U.S. toll free 888-254-3590 or international 720-543-0302, confirmation code: 7211894.

Roche submits supplemental new drug application to FDA for Venclexta plus Gazyva for previously untreated chronic lymphocytic leukaemia with co-existing medical conditions

On March 7, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported the submission of a supplemental New Drug Application to the US Food and Drug Administration (FDA) for Venclexta (venetoclax) in combination with Gazyva (obinutuzumab) in people with previously untreated chronic lymphocytic leukaemia (CLL) and co-existing medical conditions (Press release, Hoffmann-La Roche, MAR 7, 2019, View Source [SID1234534072]). The FDA is reviewing the application under the Real-Time Oncology Review pilot programme, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

"More than 20,000 people will be diagnosed with untreated chronic lymphocytic leukaemia in the US this year, and many are ineligible for intensive chemotherapy-based options," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We are encouraged that this chemotherapy-free, fixed-duration combination is being reviewed under the FDA’s Real-Time Oncology Review pilot programme, and we are working closely with the agency to bring this new option to people with previously untreated chronic lymphocytic leukaemia as quickly as possible."

Breakthrough Therapy Designation was granted based on results of the randomised phase III CLL14 study, evaluating the fixed-duration combination of Venclexta plus Gazyva, compared to Gazyva plus chlorambucil, in people with previously untreated CLL and co-existing medical conditions. The study met its primary endpoint and showed a statistically significant reduction in the risk of disease worsening or death (progression-free survival [PFS] as assessed by investigator) compared to standard-of-care Gazyva plus chlorambucil. Safety for the Venclexta plus Gazyva combination appeared consistent with the known safety profiles of the individual medicines, and no new safety signals were identified with the combination. Data from the CLL14 study will be presented at an upcoming medical meeting. The CLL14 study is being conducted in cooperation with the German CLL Study Group (GCLLSG), headed by Michael Hallek, MD, University of Cologne.

Venclexta is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the US and commercialised by AbbVie, under the brand name Venclyxto, outside of the US.

About the CLL14 study
CLL14 (NCT02242942) is a randomised phase III study evaluating the combination of fixed-duration Venclexta plus Gazyva compared to Gazyva plus chlorambucil in patients with previously untreated chronic lymphocytic leukaemia (CLL) and co-existing medical conditions. 432 patients with previously untreated CLL were randomly assigned to receive either a 12-month duration of Venclexta alongside six-month duration of Gazyva (Arm A) or six-month duration of Gazyva plus chlorambucil followed by an additional six-month duration of chlorambucil (Arm B). The primary endpoint of the study is investigator-assessed progression-free survival (PFS). Secondary endpoints include PFS assessed by independent review committee (IRC), minimal residual disease (MRD) status, overall response (OR), complete response (with or without complete blood count recovery, CR/CRi), overall survival (OS), duration of response (DOR), event-free survival (EFS), time to next CLL treatment (TTNT), and safety. The CLL14 study is being conducted in cooperation with the German CLL Study Group (GCLLSG), headed by Michael Hallek, MD, University of Cologne.

About Venclexta/Venclyxto (venetoclax)
Venclexta/Venclyxto is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other tumours, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclexta/Venclyxto blocks the BCL-2 protein and works to restore the process of apoptosis.

Venclexta/Venclyxto is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the US and commercialised by AbbVie, under the brand name Venclyxto, outside of the US. Together, the companies are committed to research with Venclexta, which is currently being studied in clinical trials across several types of blood and other cancers.

In the US, Venclexta has been granted five Breakthrough Therapy Designations by the FDA: in combination with Gazyva for people with previously untreated chronic lymphocytic leukaemia (CLL) and co-existing medical conditions; in combination with Rituxan for people with relapsed or refractory CLL; as a monotherapy for people with relapsed or refractory CLL with 17p deletion; in combination with hypomethylating agents (azacitidine or decitabine) for people with untreated acute myeloid leukaemia (AML) ineligible for intensive chemotherapy; and in combination with low-dose cytarabine for people with untreated AML ineligible for intensive chemotherapy.

About Gazyva (obinutuzumab)
Gazyva is an engineered monoclonal antibody designed to attach to CD20, a protein expressed on certain B cells, but not on stem cells or plasma cells. Gazyva is designed to attack and destroy targeted B-cells both directly and together with the body’s immune system. Gazyva is marketed as Gazyvaro in the EU and Switzerland.

Gazyva/Gazyvaro is currently approved in more than 90 countries in combination with chlorambucil for people with previously untreated chronic lymphocytic leukaemia, in more than 80 countries in combination with bendamustine for people with certain types of previously treated follicular lymphoma and in more than 70 countries in combination with chemotherapy for previously untreated follicular lymphoma.

Additional combination studies investigating Gazyva/Gazyvaro with other approved or investigational medicines, including cancer immunotherapies and small molecule inhibitors, are underway across a range of blood cancers.

About the German CLL Study Group (GCLLSG)
Founded in 1996 and headed by Michael Hallek, MD, the GCLLSG has been running various phase III, phase II and phase I trials in chronic lymphocytic leukaemia (CLL) with the goal to provide optimal treatment to patients suffering from this disease. Among those were landmark trials like the CLL8 and the CLL11 trials which led to the current standard of care in CLL. For many years, GCLLSG has been aiming to improve not just the treatment of younger and physically fit patients, but also that of elderly and less fit patients. These patients are generally underrepresented in clinical trials although they constitute the majority of CLL patients treated by doctors in daily practice. The GCLLSG is an independent non-profit research organisation supported by the German Cancer Aid (Deutsche Krebshilfe) www.dcllsg.de.

About Roche in haematology
For more than 20 years, Roche has been developing medicines that redefine treatment in haematology. Today, we are investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. In addition to approved medicines MabThera/Rituxan (rituximab), Gazyva/Gazyvaro (obinutuzumab), and Venclexta/Venclyxto (venetoclax) in collaboration with AbbVie, Roche’s pipeline of investigational haematology medicines includes Tecentriq (atezolizumab), an anti-CD79b antibody drug conjugate (polatuzumab vedotin/RG7596) and a small molecule which inhibits the interaction of MDM2 with p53 (idasanutlin/RG7388). Roche’s dedication to developing novel molecules in haematology expands beyond malignancy, with the development of Hemlibra (emicizumab), a bispecific monoclonal antibody for the treatment of haemophilia A.