On August 7, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported financial results for the quarter ended June 30, 2019, and provides an update on the Company’s recent pipeline developments, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 7, 2019, View Source [SID1234538334]).

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2019 is a transformative year for Rocket as we transition to a registrational-stage development company focused on the growth of our multi-platform gene therapy pipeline," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "This quarter, we made a lot of progress, and we now have three Rocket-sponsored gene therapy programs in the clinic, including our first AAV gene therapy program. The initiation of our Phase 1 clinical trial for Danon disease marked an important milestone for Rocket as we advanced the first AAV gene therapy for a monogenic heart failure syndrome into the clinic. With each clinical milestone, we are one step closer to rapidly providing potentially curative treatments to patients severely in need."

Recent Pipeline Developments

Two patients treated with RP-L102 "Process B" in the Phase 1 trial for Fanconi Anemia (FA) at Stanford University with initial data anticipated in six to nine months. Two patients were treated earlier this year at the Center for Definitive and Curative Medicine at Stanford University School of Medicine, the lead U.S. clinical site. "Process B" incorporates a modified cell enrichment process, transduction enhancers, and commercial-grade vector manufacturing and cell processing. The global Phase 2 study is anticipated to commence in the fourth quarter with alignment from the U.S. Food and Drug Administration (FDA). Ongoing follow-up data on patients from "Process A" and preliminary data on patients from "Process B" will be presented in the fourth quarter.
Patient dosing continues in first cohort in the Phase 1 clinical trial of RP-A501 for the treatment of Danon disease. Rocket continues to enroll patients in the trial and anticipates Phase 1 data in 2020. The study is designed to assess the safety and tolerability of a single infusion of RP-A501. Pediatric dosing will initiate pending determination of safety in a patient population comprised of older adolescents and young adults.
Commencement of Phase 1/2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Phase 1 portion of the registrational trial is underway with the first patient’s cells harvested in the second quarter. The Phase 1 portion of the trial is expected to enroll two patients and will assess the safety and tolerability of RP-L201. Preliminary data will be presented in the fourth quarter.
Anticipated Milestones

FA (RP-L102)
End-of-Phase 1 Meeting with FDA (3Q19)
Commencement of E.U. Phase 2 study (3Q19)
Commencement of U.S. Phase 2 study (4Q19)
Additional data for "Process A" (4Q19)
Initial Phase 1 data for "Process B" (4Q19)
Danon Disease (RP-A501)
Phase 1 data (2020)
LAD-I (RP-L201)
Initial Data from RP-L201 (4Q19)
PKD (RP-L301)
IMPD clearance (3Q19)
Initiation of Phase 1 study (4Q19)
IMO (RP-L401)
Initiation of clinical study (2020)
Upcoming Investor Conferences

Leerink’s Spotlight Series on Rare & Genetic Diseases—August 7, 2019 in Boston, M.A.
Citi’s 14th Annual Biotech Conference—September 4, 2019 in Boston, M.A.
Baird’s 2019 Global Healthcare Conference—September 5, 2019 in New York, N.Y.
Morgan Stanley’s 17th Annual Global Healthcare Conference—September 11, 2019 in New York, N.Y.
Oppenheimer’s Fall Summit Focused on Specialty Pharma and Rare Disease Companies—September 23, 2019 in New York, N.Y.
Ladenburg Thalmann’s 2019 Healthcare Conference—September 24, 2019 in New York, N.Y.
Second Quarter 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2019, were $257.8 million.
Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $14.0 million for the three months ended June 30, 2019, compared to $10.8 million for the three months ended June 30, 2018. The increase was primarily driven by an increase in clinical trial expenses of $2.1 million as the Phase 1 clinical trials commenced for Fanconi Anemia and Danon disease and an increase in license expense of $1.1 million.
G&A expenses. General and administrative expenses were $4.4 million for the three months ended June 30, 2019, compared to $4.1 million for the three months ended June 30, 2018.
Net loss. Net loss was $18.7 million or $0.38 per share (basic and diluted) for the three months ended June 30, 2019, compared to $15.8 million or $0.40 per share (basic and diluted) for the three months ended June 30, 2018.
Shares outstanding. 50,332,435 shares of common stock were outstanding as of June 30, 2019.
Financial Guidance

Cash position. As of June 30, 2019, we had cash, cash equivalents and investments of $257.8 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2021.

On August 7, 2019 Sierra Oncology, Inc. (SRRA), a late-stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported that Dr. Nick Glover, President and Chief Executive Officer, will present an overview of the company at 2:30 pm ET on Wednesday, August 14 at the Wedbush PacGrow Healthcare Conference being held in New York (Press release, Sierra Oncology, AUG 7, 2019, View Source [SID1234538351]).

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A live audio webcast and archive of the presentation will be accessible through the Sierra Oncology website at www.sierraoncology.com.

On August 7, 2019 Epigenomics AG (FSE: ECX, OTCQX: EPGNY, the "Company") reported the operational achievements plus financial results for the second quarter and the first half of 2019 (Press release, Epigenomics, AUG 7, 2019, View Source [SID1234538264]).

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Operational highlights

– In January 2019, the Company announced positive results from a microsimulation model for the Epi proColon blood test. The results are awaiting publication.

– In March, the bipartisan "Donald Payne Sr. Colorectal Cancer Detection Act" (HR 1765) was reintroduced into the U.S. House of Representatives. In the meantime, the law has won numerous supporters in both parties and especially in the key committees of Congress. The latter is of great importance so that the law can be voted on – if not individually, then as part of a legislative package.

– On May 3, 2019, the Centers for Medicare & Medicaid Services (CMS) accepted the Company’s application to review Epi proColon as part of a National Coverage Determination (NCD). Once the review process has been opened, there is a statutory timeframe of maximum nine months within which CMS must have reached a decision and Epigenomics therefore has clarity regarding the reimbursement of Epi proColon.

6M 2019 Financial results
– In the first half of 2019, product revenue increased by 84% year-on-year to EUR 660 thousand.

– License revenue decreased to EUR 19 thousand (6M 2018: EUR 413 thousand) due to the termination of the collaboration with the Chinese licensing partner.

– Total revenue in the first six months of 2019 decreased from EUR 771 thousand to EUR 679 thousand compared to the same period in the prior year due to lower license revenue, which were not fully offset by higher product revenue.

– Research and development costs increased from EUR 3,043 thousand in the previous year to EUR 3,867 thousand. This increase resulted from expenses related to the post-approval study for Epi proColon and the HCC (liver cancer test) study in the U.S.

– Selling and administrative expenses rose by from EUR 3,883 thousand to EUR 4,859 thousand in the reporting period, due to an increase in sales and marketing activities related increased product revenue and commercial preparation of reimbursement coverage.

– Overall, operating costs were lower than estimated for the first six months of 2019. Operating expenses increased from EUR 7.1 million to EUR 9.4 million compared to prior year for the abovementioned reasons.

– Adjusted EBITDA (before share-based payment expenses) was EUR -7.2 million in the first six months of 2019 compared to EUR -5.4 million for the same period prior year.

– The net loss for the period was EUR -7.4 million vs. EUR -5.8 million for the prior year; the loss per share decreased to EUR 0.21 (6M 2018: EUR 0.24) due to the higher number of outstanding shares following the capital increase carried out in the second half of 2018.

– Cash consumption from operating activities increased by EUR 3.6 million to EUR 7.8 million in the first half of 2019 due to higher operating costs.

– As of June 30, 2019, liquidity amounted to EUR 9.1 million (including marketable securities) compared to EUR 17.1 million at year-end 2018.

Greg Hamilton, CEO of Epigenomics AG: "We continue to make progress on CMS reimbursement. CMS’s acceptance of our NCD is an important milestone. While the process of opening the NCD is taking longer than we would like we still remain optimistic and will continue to pursue all available paths to achieve coverage for Epi proColon."

Outlook 2019: revenue forecast adjusted
Revenue

– Due to the delays in the reimbursement decision in the U.S. described above, the revenue forecast has been adjusted from between EUR 3.0 million and EUR 6.0 million to between EUR 2.0 million and EUR 4.0 million.

EBITDA / Cash consumption

– Epigenomics AG specifies its guidance for adjusted EBITDA (before share-based payment expenses). For the full year 2019, the Company expects an adjusted EBITDA within the range of EUR -12.5 million to EUR -14.0 million. Cash consumption is expected to be within a range of EUR -13.5 million to EUR -15.0 million, due to payments in the first quarter of 2019 for which the corresponding expenses had already been incurred in 2018.

Further information
The financial report for the first six months of 2019 is available on the Epigenomics website: View Source." target="_blank" title="View Source." rel="nofollow">View Source

Conference call for analysts and investors
Epigenomics AG will host a conference call for analysts and investors today at 4.00 pm (CET) / 10.00 am (EDT). The webcast can be accessed on the Company’s website: View Source
The dial-in numbers for the conference call are:
Dial-in number Germany: +49 30 232 531 411
Dial-in number UK: +44 1635 598 060
Dial-in number USA: +1 516-269-8983
Participants are kindly asked to dial in 10 minutes prior to the start of the call.
An audio replay of the conference call will be provided on the Epigenomics’ website subsequently.

On August 7, 2019 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported second quarter 2019 financial results (Press release, CytomX Therapeutics, AUG 7, 2019, View Source/news-releases/news-release-details/cytomx-therapeutics-announces-second-quarter-2019-financial" target="_blank" title="View Source/news-releases/news-release-details/cytomx-therapeutics-announces-second-quarter-2019-financial" rel="nofollow">View Source [SID1234538301]).

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As of June 30, 2019, CytomX had cash, cash equivalents and short-term investments of $349.1 million.

"CytomX continues to make progress across its pipeline. Highlights of the second quarter included additional presentations of clinical data for our lead, wholly owned assets, CX-072 and CX-2009, which further demonstrated the potential of these two novel anti-cancer agents," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "Our clinical work to date with our lead programs provides validation for our unique approach to targeting antibody therapies to the tumor microenvironment and, accordingly, the discovery and development of new and differentiated treatments for cancer patients."

Business Highlights and Recent Developments

PROCLAIM-CX-072 (PD-L1 Probody Therapeutic) Clinical Program

CX-072 is a wholly owned Probody therapeutic targeting PD-L1, a clinically and commercially validated anti-cancer target.
CytomX presented updated clinical data from monotherapy expansion cohorts (Part D) of the PROCLAIM-CX-072 Phase 1/2 study, evaluating the safety and efficacy of CX-072 in multiple tumor types at 10 mg/kg at the 2019 Annual Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. These data demonstrated a favorable safety profile and evidence of anti-cancer activity in certain patients with triple negative breast cancer, anal squamous cell carcinoma, cutaneous squamous cell carcinoma and undifferentiated pleomorphic sarcoma.
David Page, M.D., Medical Oncologist, Providence Cancer Center presented clinical data from PROCLAIM-CX-072 monotherapy and in combination with ipilimumab (YERVOY) as part of a Poster Discussion Session at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting.
PROCLAIM-CX-2009 (CD166 Probody Drug Conjugate) Clinical Program

CX-2009 is a wholly owned, first in class Probody drug conjugate (PDC) targeting CD166, a novel antigen that is broadly and highly expressed in many types of cancer.
CytomX reported preliminary data from the dose-escalation phase (Part A and A2) of the ongoing PROCLAIM-CX-2009 Phase 1/2 study, evaluating the safety and antitumor activity of CX-2009 in seven selected tumor types, at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. CX-2009 was generally well tolerated. Single agent anti-cancer activity was observed in certain patients with breast cancer, ovarian cancer and head and neck cancer.
CX-2029 (CD71 Probody Drug Conjugate) Clinical Program

CX-2029 is a first in class PDC targeting CD71, the Transferrin Receptor, a highly efficient cellular mechanism for the internalization of antibody drug conjugates in preclinical models.
CD71 is widely expressed on normal tissues and therefore is considered to be an undruggable clinical target for conventional antibody drug conjugate technology.
CytomX discovered and is developing CX-2029 in collaboration with AbbVie to potentially turn CD71 into a druggable target.
CytomX continues to enroll patients in the PROCLAIM-CX-2029 Phase 1/2 study evaluating CX-2029 as monotherapy in patients with solid tumors or lymphomas.
BMS-986249 (CTLA-4 Probody Therapeutic) Clinical Program

Bristol-Myers Squibb (BMS) continues enrollment in a Phase 1/2 dose escalation clinical study evaluating BMS-986249 alone and in combination with OPDIVO (nivolumab) in solid tumors that are advanced and have spread.
BMS is preparing to initiate the Phase 2 portion of this clinical trial, upon which CytomX is entitled to a $10 million milestone payment.
AbbVie Second Target Selection Under Strategic Oncology Collaboration

In July 2019, CytomX announced its partner AbbVie selected a second target under the companies’ 2016 Discovery Collaboration and Licensing Agreement to discover and develop Probody drug conjugates. The target selection triggered a $10 million payment to CytomX.
Second Quarter 2019 Financial Results
Cash, cash equivalents and short-term investments totaled $349.1 million as of June 30, 2019, compared to $436.1 million as of December 31, 2018. The decrease of $87.0 million for the six months ended June 30, 2019 included certain infrequent payments such as $5.0 million for the acquisition from an Astellas subsidiary of technical know-how related to drug conjugate linker-toxin and CD3-based bispecific antibody technology in the first quarter, a $13.7 million federal tax payment for the 2018 tax return filing in the second quarter and approximately $4.7 million related to the UCSB license agreement, also in the second quarter.

Revenue was $9.0 million for the three months ended June 30, 2019, compared to $21.3 million for the three months ended June 30, 2018. The decrease in revenue of $12.3 million for the three months ended June 30, 2019 compared to the corresponding period in 2018 was primarily due to the $21.0 million milestone payment (net of the associated sublicense fee of $4.0 million) earned in May 2018 under the CD71 Agreement with AbbVie, of which $9.9 million was recognized in the second quarter of 2018 reflecting the percentage completed-to-date of the project related to this milestone.

Research and development expenses increased $5.3 million during the three months ended June 30, 2019 compared to the corresponding period in 2018. The increase was attributable to $3.4 million in license fees and maintenance fees related to an amendment to the UCSB Licensing Agreement (which included the issuance of 150,000 shares of Company common stock valued at $1.6 million, an upfront payment of $1.0 million and an additional annual maintenance fee of $0.8 million), an increase of $0.8 million sublicense expense pertaining to the $10.0 million milestone payment earned upon the AbbVie selection of the second target in the second quarter of 2019 under the Amended and Restated Discovery Collaboration and License Agreement, an increase of $2.4 million in personnel-related expenses due to an increase in headcount, an increase of $0.5 million in clinical related expenses resulting from increased clinical trial activities and an increase of $0.7 million in the allocation of information technology and facilities related expenses resulting from an increase in headcount, partially offset by a decrease of $2.3 million in laboratory contracts and services as a result of timing of manufacturing activities.

General and administrative expenses increased by $0.4 million during the three months ended June 30, 2019 compared to the corresponding period in 2018. The increase was attributable to an increase of $1.0 million in personnel-related expenses due to an increase in headcount, an increase of $0.3 million for dues and subscriptions primarily related to software and other IT services and an increase of $0.2 million in professional service expenses, partially offset by a decrease of $0.5 million in consulting and contract services and a decrease of $0.7 million through increased expense allocation of information technology and facilities-related expenses to research and development due to an increase in research and development headcount.

Teleconference Scheduled Today at 5:00 p.m. ET
Conference Call/Webcast Information

CytomX management will host a conference call today at 5:00 p.m. ET. Interested parties may access the live audio webcast of the teleconference through the "Investor & News" section of CytomX’s website at View Source or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) and using the passcode 7785617. An archive of the webcast will be available on the CytomX website from August 7, 2019, until August 21, 2019.

On August 7, 2019 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported that The University of New Mexico (UNM) Comprehensive Cancer Center and the New Mexico Alliance for Cancer Care will utilize Personalis’ cancer immunogenomics platform, ImmunoID NeXT, for the investigation of biomarkers of response to a novel treatment paradigm in women with ovarian cancer (Press release, Personalis, AUG 7, 2019, View Source [SID1234538335]). The basis of this trial, which evaluates the combination of Olaparib and Tremelimumab in women with recurrent BRCA-deficient ovarian cancers, is based on the work of Sarah Adams, MD, that indicated that immune priming with targeted cytotoxic therapy using a PARP-inhibitor can sensitize ovarian tumors to immune therapy and optimize patient survival. The clinical trial is being conducted at several ORIEN centers across the United States. For more information please visit clinicaltrials.gov identifier NCT02571725.

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"We are delighted to collaborate with Personalis on this study," said the principal investigator of the study, Dr. Adams, Associate Professor, Division of Gynecologic Oncology at the UNM Comprehensive Cancer Center. "Comprehensive immuno-profiling will inform on biomarkers of response for this experimental treatment in women with BRCA1 or BRCA2 germline mutated ovarian cancers, for which there aren’t standard curative measures."

Personalis ImmunoID NeXT Platform provides a comprehensive interrogation and analysis of ~20,000 genes in both DNA and RNA. The platform is an end-to-end solution for immuno- and precision oncology biomarker discovery applications, simultaneously enabling the analysis of: tumor escape mechanisms (including HLA typing and somatic mutation detection), immune repertoire profiles, neoantigen load, tumor mutational burden (TMB), microsatellite instability (MSI), oncoviruses, and immune checkpoint gene expression.