On May 02, 2016 Aduro Biotech, Inc. (NASDAQ:ADRO) reported financial results for the first quarter 2016. For the three months ended March 31, 2016, net loss was $28.8 million, or $0.45 per share, compared to a net loss of $16.6 million, or $39.97 per share for the same period in 2015 (Press release, Aduro BioTech, MAY 2, 2016, View Source;p=RssLanding&cat=news&id=2163866 [SID:1234511796]).

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Cash and cash equivalents and marketable securities totaled $408.5 million at March 31, 2016, compared to $431.0 million at December 31, 2015.

"Our cash position remains strong and we are uniquely positioned in the immunotherapy field with a deep pipeline of assets and programs," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "We anticipate reaching key inflection points this year for our pancreatic cancer program, with data from our Phase 2b ECLIPSE clinical trial this quarter and interim results from the Phase 2b STELLAR trial in the second half of the year."

Recent Progress

Initiated Phase 1/2 combination clinical trial of CRS-207 and epacadostat in ovarian cancer
Established the Immunotherapeutics and Vaccine Research Initiative with UC Berkeley
Announced receipt of $22.4 million in clinical development milestone payments from Janssen
Expanded patent portfolio with newly issued patents covering LADD and GVAX combination therapy

First Quarter 2016 Financial Results
Revenue was $4.0 million for the first quarter of 2016, compared to $9.6 million for the same period in 2015. The decrease was primarily due to deferred revenue from our Janssen collaborations.

Research and development expenses were $20.9 million for the first quarter of 2016, compared to $10.6 million for the same period in 2015. This increase was primarily due to manufacturing costs, clinical development expenses associated with our ongoing trials in pancreatic cancer, ovarian cancer and mesothelioma, as well as personnel related expenses associated with continued workforce growth.

General and administrative expenses were $9.0 million for the first quarter of 2016, compared to $6.2 million for the same period in 2015. This increase was primarily due to stock based compensation expense and additional personnel and facility related expenses to support the company’s growth.

Loss from remeasurement of fair value of warrants was zero for the first quarter of 2016, compared to $9.3 million for the same period in 2015. In April 2015, all such warrants ceased being liability-classified as the contingency surrounding the number of shares issuable upon the warrant exercise expired. In April 2015, all outstanding warrants were equity-classified and not subject to future remeasurement.

Provision for income taxes was $3.2 million for the first quarter of 2016, compared to zero for the same period in 2015. The income tax expense recorded for the first quarter of 2016 primarily related to current and deferred federal income taxes.

On May 2, 2016 Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported financial results for the first quarter ended March 31, 2016 and provided a summary of recent corporate highlights (Press release, Regulus, MAY 2, 2016, View Source [SID:1234511759]).

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"2016 is shaping up to be a pivotal year of growth for Regulus. Our progress in the first quarter has continued to demonstrate our ability to advance multiple clinical programs and execute well on our stated goals," said Paul Grint, M.D., President and CEO of Regulus. "RG-101, with curative potential for hepatitis C viral infection, remains our primary focus and we are very gratified with the efficacy and safety reported thus far. The unique mechanism of action for RG-101, targeting a human factor necessary for viral replication within the liver, supports the potential of RG-101 to become a backbone agent for combinations with all classes of direct anti-HCV therapies. In the coming quarters, we look forward to accelerating our broad Phase II development program and reporting additional data that will help define the regulatory pathway for potential approval."

First Quarter 2016 Financial Results & Highlights

Regulus reported a net loss of $21.2 million for the quarter ended March 31, 2016, compared to a net loss of $14.5 million for the quarter ended March 31, 2015. Basic and diluted net loss per share was $0.40 for the quarter ended March 31, 2016, compared to $0.29 for the quarter ended March 31, 2015.

Regulus recognized revenue of $0.5 million for the quarter ended March 31, 2016, compared to $4.2 million for the quarter ended March 31, 2015. Revenue for the quarter ended March 31, 2016 consisted of amortization of up-front payments from Regulus’ strategic alliances and collaborations, which are recognized over the estimated period of performance. Revenue for the quarter ended March 31, 2015 included $3.1 million in milestones and other payments earned under our collaboration with AstraZeneca.

Research and development expenses were $16.8 million for the quarter ended March 31, 2016, compared to $13.4 million for the quarter ended March 31, 2015. This increase was primarily driven by Phase II clinical trial costs for RG-101 and an increase in ongoing clinical trial costs associated with our global ATHENA natural history of disease study and manufacturing costs for RG-012.

General and administrative expenses were $5.1 million for the quarter ended March 31, 2016, compared to $3.6 million for the quarter ended March 31, 2015. This increase was primarily driven by non-cash stock-based compensation.

As of March 31, 2016, Regulus had $106.0 million in cash, cash equivalents and short-term investments, including restricted cash of $0.9 million, and 52,774,550 shares of common stock outstanding.

Recent Highlights

RG-101 (GalNAc-conjugated anti-miR122 for the treatment of Hepatitis C Virus)

Reported Interim Results at ILC 2016. At the International Liver Congress Meeting (ILC 2016), Regulus announced interim results during an oral presentation in the general session from one of the company’s ongoing Phase II studies of RG-101 for the treatment of HCV. The study was designed to evaluate a shortened, four-week treatment regimen containing a subcutaneous administration of 2 mg/kg of RG-101 at Day 1 and Day 29, in combination with 4 weeks of once/daily approved anti-viral agents Harvoni, Olysio, or Daklinza. The study enrolled 79 treatment naïve genotype 1 and 4 HCV patients (Harvoni arm, n=27, Olysio arm, n=27, Daklinza arm, n=25). Available data from 64 patients at the interim analysis demonstrated high virologic response rates in all treatment groups, providing further evidence of RG-101’s potential to successfully shorten the duration of oral regimens. To date, RG-101 has been generally well tolerated with the majority of adverse events considered mild or moderate, and with no study discontinuations. The primary endpoint analysis (12 week follow up) for all 79 patients in the study are anticipated to be reported in late Q2 2016.
Initiated Phase II Combination Study with GSK. In accordance with Regulus’ clinical trial collaboration with GSK, the companies have initiated a Phase II clinical trial evaluating the combination of RG-101 and GSK2878175, an oral, non-nucleoside NS5B polymerase inhibitor, in HCV patients. In parallel, GSK is working to develop a long-acting parenteral ("LAP") formulation of GSK2878175 as a single intra-muscular injection, providing the potential for a single-visit therapeutic treatment for HCV. Regulus anticipates reporting interim safety and efficacy data from this study by the end of 2016.
Enrollment Initiated in US Phase I Study. Today, Regulus announced that enrollment is near completion in a multi-center, open label, non-randomized Phase I study to compare the safety, tolerability, pharmacokinetics, and pharmacodynamics of RG-101 in subjects with severe renal insufficiency or end-stage renal disease ("ESRD") to healthy control subjects, and further explore RG-101 in hepatitis C infected subjects with severe renal insufficiency or ESRD. Regulus anticipates reporting safety and efficacy data from the HCV/severe renal impairment or ESRD arm in the second half of 2016.
RG-012 (anti-miR21 for the treatment of Alport syndrome)

Advanced ATHENA Natural History Study. Regulus continues to advance the ATHENA natural history study in patients with Alport syndrome and anticipates reporting initial observations in the second quarter of 2016. Longitudinal data obtained in the ATHENA natural history study will help inform the design of the Phase II study.
Phase II Study. Planning is underway to initiate the Phase II study by mid-2016.
Multiple RG-012 Poster Presentations Have Been Accepted for the ERA-EDTA 53rd Congress in Vienna, AUS in May 21-24, 2016. Poster presentations will include preclinical data and longitudinal data obtained in the Athena natural history of disease study.
Additional Highlights

Advanced Preclinical Portfolio. Regulus advanced several programs against undisclosed microRNA targets, and is on track to nominate a fourth candidate for clinical development by YE 2016.
Regulus to Present at Multiple Upcoming Healthcare Conferences. Upcoming presentations include the Bank of America Merrill Lynch 2016 Health Care Conference, May 10-13, 2016 in Las Vegas, the UBS 2016 Global Healthcare Conference, May 23-25 in New York City and the Jeffries 2016 Healthcare Conference, June 7-10, 2016 in New York City.

On May 2, 2016 CEL-SCI Corporation (NYSE MKT: CVM) ("CEL SCI" or the "Company") reported that during the month of April it has enrolled 41 patients in its ongoing Phase 3 trial of its investigational immunotherapy Multikine* (Leukocyte Interleukin, Injection) in patients with advanced primary head and neck cancer (Press release, Cel-Sci, MAY 2, 2016, View Source [SID:1234511755]). This is a new monthly enrollment record, surpassing the prior record of 38 patients enrolled in October 2015. Total patient enrollment for the trial is now 797 as of April 30, 2016.

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About the Multikine Phase 3 Study

The Multikine Phase 3 study is enrolling patients with advanced primary (not yet treated) squamous cell carcinoma of the head and neck. The objective of the study is to demonstrate a statistically significant improvement in the overall survival of enrolled patients who are treated with the Multikine treatment regimen plus standard of care ("SOC") vs. subjects who are treated with SOC only.

About Multikine

Multikine (Leukocyte Interleukin, Injection) is an investigational immunotherapeutic agent that is being tested in an open-label, randomized, controlled, global pivotal Phase 3 clinical trial as a potential first-line treatment for advanced primary squamous cell carcinoma of the head and neck. Multikine is designed to be a different type of therapy in the fight against cancer: one that appears to have the potential to work with the body’s natural immune system in the fight against tumors.

Multikine is also being tested in a Phase 1 study under a Cooperative Research and Development Agreement ("CRADA") with the U.S. Naval Medical Center, San Diego, and at University of California, San Francisco (UCSF), as a potential treatment for peri-anal warts in HIV/HPV co-infected men and women. Dr. Joel Palefsky, a world-renowned scientist and Key Opinion Leader (KOL) in human papilloma virus (HPV) research and the prevention of anal cancer, is the Principal Investigator at UCSF, which was added to the study in July 2015.

CEL-SCI has also entered into two additional co-development agreements for up to $3 million each with Ergomed Clinical Research Limited to further the development of Multikine for cervical dysplasia/neoplasia in women who are co-infected with HIV and HPV and for peri-anal warts in men and women who are co-infected with HIV and HPV.

On May 2, 2016 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company with three clinical-stage development programs focused on the emerging field of regenerative medicine, reported that clinical data from its AST-VAC1 (antigen-presenting autologous dendritic cells) immunotherapy clinical program will be presented during an oral session at the upcoming American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 19th Annual Meeting, to be held on May 4-7, 2016 in Washington, D.C (Press release, BioTime, MAY 2, 2016, View Source;p=RssLanding&cat=news&id=2163621 [SID:1234511741]).

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Jane S. Lebkowski, Ph.D., Asterias’ President of R&D and Chief Scientific Officer, will present the data, which is from the Company’s AST-VAC1 Phase 2 clinical trial in acute myelogenous leukemia (AML) and was first presented at the 2015 annual meeting of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper). The abstract is now available online on the ASGCT (Free ASGCT Whitepaper) Annual Meeting website.

Presentation Details

Title: Long-Term Relapse-Free Survival of Patients with Acute Myeloid Leukemia (AML) Receiving a Telomerase-Engineered Dendritic Cell Immunotherapy
Abstract Number: 276
Session Title: Cancer-Immunotherapy, Cancer Vaccines I
Date: Thursday, May 5, 2016
Time: 5:00 – 5:15 PM
Room: Washington 4

On May 2, 2016 AVEO Oncology (NASDAQ:AVEO) reported that it has filed provisional patent applications with the United States Patent and Trademark Office (USPTO) covering composition of matter claims for AV-353, the Company’s potent inhibitory antibody specific to Notch 3 (Press release, AVEO, MAY 2, 2016, View Source;p=RssLanding&cat=news&id=2163617 [SID:1234511738]). These patent applications are the second set of applications related to AV-353 and the Company’s Notch 3 antibody program.

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The Notch 3 signaling pathway is important in cell-to-cell communication involving gene regulation mechanisms that control multiple cell differentiation processes during the entire life cycle. Scientific literature has implicated the Notch 3 receptor pathway in multiple diseases, including cancer, cardiovascular diseases and neurodegenerative conditions. Recent publications, including Nature Medicine (2009), have implicated the Notch 3 pathway in Pulmonary Arterial Hypertension (PAH). PAH is a rare and life-threatening disorder that affects approximately 250,000 people worldwide and is caused by enlargement of the arterial walls in small arteries between the heart and the lungs, resulting in restricted blood flow.

Current treatments in PAH focus only on controlling symptoms by avoiding vasoconstriction and increasing vasodilation of vessels and do not reverse the underlying cause of the disease. In contrast, with the results of a recently concluded research study supported by AVEO, AV-353 has generated a growing body of preclinical data that supports AV-353’s ability to potentially reverse the disease phenotype, which would represent a potential disease-modifying approach to treatment. A manuscript of the results is being prepared for submission to a peer-reviewed journal.

"Like AV-380 in cachexia, which AVEO licensed to Novartis in 2015, AV-353 is an AVEO legacy discovery program that, with the appropriate development support from a partner, has the potential to transform the treatment paradigm for a debilitating disorder and underserved patient population," said Michael Bailey, president and chief executive officer of AVEO. "We believe that AV-353’s selectivity and high affinity to Notch 3, as demonstrated in preclinical studies, makes it unique relative to pan-notch inhibitory or ligand inhibitory approaches in PAH treatment, allowing us to potentially build a broad patent estate around this program. Consistent with our current focus on developing oncology therapeutics, AVEO is currently seeking an appropriate partner to develop and commercialize AV-353 worldwide in PAH."