On May 03, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported that it has completed the initial dosing cohort in its SL-801 Phase 1 advanced solid tumor trial (Press release, Stemline Therapeutics, MAY 3, 2016, View Source [SID:1234511819]). The second cohort is currently open. SL-801 is a novel, oral, small molecule that reversibly inhibits the XPO1 (Exportin-1) nuclear transport protein, a clinically validated target active in a wide variety of cancer types.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This Phase 1 dose escalation trial will enroll up to 50 patients with advanced solid tumors at multiple centers in the U.S. The trial is designed to evaluate safety, identify an optimal dosing regimen, and detect potential signals of efficacy in one or more cancer types.

Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, stated, "We are very pleased to have dosed the first patients in our SL-801 program during the first quarter, in-line with our expectations, and recently cleared the first dosing cohort. We are continuing to enroll patients with a wide range of solid cancer types in this trial in order to identify specific indications with both sensitivity to the agent as well as viable registration pathways. We believe we can leverage the unique properties of this compound along with our knowledge of the XPO1 space to create significant value from the program early on."

On May 3, 2016 Kancera AB reported their interim results for Q1 2016, January 1 – March 31, 2016 (Press release, Kancera, MAY 3, 2016, View Source;releaseID=1143395 [SID:1234511821]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The period January to March 2016 in brief

R&D expenses for the period amounted to SEK 4.2m (SEK 4.2m).
Operating income for the period amounted to SEK -5.1m (SEK 4.9m).
Income after financial items for the period amounted to SEK -5.0m (SEK -4.9m).
Earnings per share for the period were SEK -0.05 (SEK -0.05).
Cash flow from operating activities for the period amounted to SEK -6.3m (SEK -5.1m).
Equity as of March 31, 2016 amounted to SEK 17.0m (SEK 22.6m) or SEK 0.16 (SEK 0.22) per share. The equity/assets ratio as of March 31, 2016 was 70 percent (71 percent).
Cash and cash equivalents as of March 31, 2016 amounted to SEK 12.1m (SEK 17.8m).
Significant events during the period

Kancera has from the 1st of January 2016 extended the lease of the company’s laboratories within the Karolinska Science Park for three years through an agreement with Humlegården Fastigheter.
Kancera has provided an update of the small molecule patent portfolio.
– A patent covering small molecule PFKFB3 inhibitors has been approved in the USA.

– A patent application covering new chemical series in the HDAC6 project has been filed.

– An international patent application covering ROR inhibitors has been strengthened by adding examples of additional highly potent ROR inhibitors.

Kancera reported that the company has developed a new series of ROR inhibitors that show improved pharmaceutical properties which will allow preclinical studies of their effect on e.g. solid tumors. These results have prompted Kancera to concentrate the investments in the ROR project to small molecule inhibitors and terminate the product development of a ROR-based vaccine. Furthermore, Kancera reported results from the Fractalkine project showing that KAN0440567 after oral administration to mice effectively blocks the function of the Fractalkine receptor.
Kancera announced that the company according to plan has received another payment of about SEK 2.8 million in January, 2016 from the EU for the A-PARADDISE project, which aims to develop drugs against parasitic diseases.
Significant events after the end of the reporting period

Kancera announced that the Extraordinary General Meeting of Kancera AB approved the Board’s decision of 6 April 2016 to issue new shares and warrants in the form of units. The decision includes a preferential rights issue of shares and warrants (units) ("New Share Issue"), which upon full subscription brings Kancera about SEK 52 million before issue expenses and with a full exercise of the warrants brings Kancera an additional SEK 52-62 million. The issue assets will be used for Kancera’s drug development, clinical studies and the further development of the Company’s capacity to commercialize products. The majority of Kancera’s resources are now concentrated on taking at least one of Kancera’s drug candidates in the ROR and Fractalkine projects to clinical trial for chronic lymphocytic leukemia and pancreatic cancer, respectively. In parallel, the Company intends to validate a broader use of the drug candidates from these projects in order to demonstrate their full commercial potential.
Kancera reported that ROR inhibitors have been tested against human triple negative breast cancer transferred to zebra fish. The experiments showed that Kancera’s small molecule ROR inhibitors are able to both reduce tumor size and metastases (spread) of this aggressive tumor form. Further, Kancera reported that the company´s PFKFB3 inhibitors are active in the same model of triple negative breast cancer and that a patent application has been filed covering the discovery that PFKFB3 inhibitors enhance the effect of radiation treatment
Kancera reported that the Company due to positive efficacy data in disease models of cancer and pain has decided to exercise the exclusive option to acquire the Fractalkine project. The acquisition will be carried out in connection with the completion of the ongoing transfer of results and know-how from Acturum and AstraZeneca to Kancera. Payment for the project to Acturum Life Science AB will be made into three steps by a total of 6 million shares, of which the first payment is due at the submission of the application for authorization of a clinical trial after an approval by Kancera´s shareholders. In parallel, the company intends to validate a broader use of the drug candidate (KAN0440567) in order to demonstrate its full commercial potential.
Statement from the CEO

In 2016, we have reported progress in the development of a new generation of ROR inhibitors with properties that we expect will enable effect against both lymphoma and solid tumors. A first study where human triple negative breast cancer was transplanted into zebra fish, shows that ROR inhibitors are able to both inhibit tumor growth and prevent spread. Further, we have been able to validate that Kancera PFKFB3 inhibitor is able to inhibit tumor growth in the same model.

On the patent side, we have completed an international patent application in the ROR project with the addition of approximately 100 selected potent compounds. Also, a patent application covering PFKFB3 substances has been approved in the United States and a new application covering the discovery that Kancera’s PFKFB3 inhibitor counteract cancer by enhancing the effect of radiation treatment has been submitted. An additional patent application has been filed in the HDAC6 project covering a new class of active substances which together with the first patent application in the project, broadens the protection around our unique selective and effective compounds against myeloma.

Furthermore, due to positive efficacy data in disease models of cancer and pain, the Kancera Board has decided to exercise the exclusive option to acquire the Fractalkine project.

The goal for the next 18-24 months is to take at least one of Kancera’s drug candidates from the Fractalkine and ROR projects to clinical trials and thus first clinical use (chronic lymphocytic leukemia/ pancreas cancer). In parallel, we also complete the evaluation of a broader use of the drug candidates from these projects in order to reduce the risk in the product development and to identify full commercial potential of the projects. The operational objectives also include delivering drug candidates from the HDAC6 and PFKFB3 projects.

Given these ambitious goals, Kancera’s Board supported by the decision of the Extraordinary General Meeting on April 22, 2016, decided to launch a new share issue. The decision includes a preferential rights issue of shares and warrants (units) which upon full subscription brings Kancera about SEK 52 million before issue costs.

With eight patent families, which together make up the intellectual property protection for four small-molecule cancer projects, we expect that Kancera has good opportunities to translate scientific results into commercially attractive new drugs against cancer. However, the development of new drugs is biologically and technically risky and challenged by international competition.

This share issue aims to push Kancera’s cancer project from a promising development phase to completion in order to be tested in cancer patients. I am convinced that we will succeed in this work.

On May 4, 2016 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH) reported its revenues and cash position for the first three months of 2016 (Press release, Innate Pharma, MAY 3, 2016, View Source [SID:1234511888]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Cash, cash equivalents and financial instruments of the Company amounted to €255.8 million as of March 31, 2016, including short term investments (€51.2m) and non-current financial instruments (€37.5m). At the same date, financial liabilities amounted to €3.6 million.

Revenues for the first three months of 2016 amounted to €5.7 million (€0.4 million for the same period in 2015). This revenue results from Innate Pharma’s collaboration and licensing agreement with Bristol-Myers Squibb and co-development and commercialization agreement with AstraZeneca:

€5.5 million resulting from the agreement with AstraZeneca, corresponding to the recognition over the period of the initial payment received in June 2015;
€0.2 million relating to the agreement with Bristol-Myers Squibb resulting from the recognition over the period of the upfront payment received in July 2011.
In 2015, revenue for the first three months resulted mainly from the recognition of the upfront payment from the agreement with Bristol-Myers Squibb.

Hervé Brailly, Chief Executive Officer and co-founder of Innate Pharma, commented: "During the first quarter of 2016, our pipeline of innovative immuno-oncology candidates continued to progress and expand. At the AACR (Free AACR Whitepaper) 2016 Annual Meeting, we presented data supporting the development of our clinical and preclinical programs, including two new programs targeting the tumor microenvironment. We are proud of these programs which further demonstrate our unique positioning in immuno-oncology.

At the same time, our most advanced programs moved forward in their clinical development. A Phase I clinical trial was started by AstraZeneca that is testing our first-in-class NKG2A checkpoint inhibitor, monalizumab, in combination with durvalumab (AstraZeneca/ MedImmune’s PD-L1 inhibitor). This fifth trial completes the roll-out of the initial clinical plan, due to start reading out in 2017. With regards to lirilumab, our first-in-class anti-KIR checkpoint inhibitor, the DSMB completed its sixth assessment of the Phase II EffiKIR study in March and recommended continuation of the trial without modification. Data on the primary endpoint are expected in the second half of 2016."

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.