On May 30, 2017 HedgePath Pharmaceuticals, Inc. (OTCQX:HPPI), a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative therapeutics for patients with cancer, reported the grant of a Type-C Guidance Meeting Request by the U.S. Food and Drug Administration (FDA) concerning further guidance from FDA for HPPI’s ongoing, open-label Phase 2(b) clinical trial studying the effect of SUBA-Itraconazole (SUBA-Cap) oral capsules in patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS), also known as Gorlin Syndrome (Press release, HedgePath Pharmaceuticals, MAY 30, 2017, View Source [SID1234519323]).

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Included in HPPI’s meeting request were summary data for 35 patients enrolled in the trial relating to reduction in target tumor burden, safety and time on study, along with specific questions to FDA regarding further steps necessary for completion of the study and reporting of final data. HPPI noted in its meeting request to FDA that all patients on SUBA-Cap therapy had some degree of measurable target tumor burden decrease with a median time on study of 32 weeks and a dropout rate of only 11%.
As a result of FDA granting HPPI’s meeting request, HPPI is required to file a complete background package for its Phase 2(b) trial results to FDA by mid-June 2017, and FDA has indicated its goal is to provide a written response to HPPI with further guidance before the end of July 2017.
Nicholas Virca, President and CEO of HPPI, stated that, "We reported to FDA that 37% of our patients in our Phase 2(b) trial have demonstrated an equal to or greater than 30% reduction in target tumor burden and there has been a complete disappearance of 28% of all target lesions across all subjects. We are testing SUBA-Cap therapy in BCCNS patients with a significant history of BCC surgeries and intend to further note in our background package that, for the 35 patients being dosed in our trial, the mean number of prior BCCs removed by surgery was 195 per patient, yet 97% of our study group have avoided surgery while on SUBA-Cap therapy. We are very pleased with these results and look forward to FDA’s feedback as we move towards the conclusion and reporting of the results of this trial."
While these data appear to be predictive of the desired final study results while HPPI seeks further guidance from FDA, readers are cautioned that no assurances can be given that (i) the final study results will match these latest results or (ii) the study when and if completed will achieve its primary and secondary endpoints or (iii) that the study will be found by FDA to be sufficient for the filing of a New Drug Application (NDA) or (iv) if an NDA is filed, that it will be approved by FDA. Further, HPPI is not committing to providing further interim updates prior to the reporting of the final study results.
About BCCNS
BCCNS results from a genetic mutation which causes the Hedgehog pathway (a major regulator of processes in cells) to function improperly, leading to the chronic formation of basal cell tumors, including potentially disfiguring lesions on the face. Industry sources estimate that there are approximately 10,000 patients in the United States with BCCNS, which has qualified SUBA-Itraconazole under the FDA’s Orphan Drug Designation Program.
About SUBA-Itraconazole
SUBA-Itraconazole is a patented and proprietary itraconazole formulation that enhances the absorption of itraconazole to improve the bioavailability of orally administered drugs that are poorly soluble. The U.S. rights to SUBA-Itraconazole for the treatment of cancer are exclusively licensed to HPPI by an affiliate of Mayne Pharma Group Limited. SUBA-Itraconazole was developed to improve absorption and significantly reduce variability compared to generic itraconazole. These benefits provide enhancements to patients and prescribers with reduced intra- and inter-patient variability, enabling a more predictable clinical response and a reduction in the active drug quantity to deliver the required therapeutic blood levels.

On May 30, 2017 Heat Biologics, Inc. ("Heat") (NASDAQ: HTBX), a leader in the development of novel therapies designed to activate a patient’s immune system against cancer, reported that it will present a poster from the Phase II trial evaluating HS-410 (vesigenurtacel-L) in combination with standard of care, Bacillus Calmette-Guérin (BCG), in the treatment of non-muscle invasive bladder cancer (NMIBC) at the 2017 ASCO (Free ASCO Whitepaper) (American Association of Clinical Oncologists) Annual Meeting at McCormick Place in Chicago, Illinois on Sunday, June 4th, 2017 (Press release, Heat Biologics, MAY 30, 2017, View Source [SID1234519321]).

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Poster Presentation Details

Title: Immune response results from vesigenurtacel-l (HS-410) in combination with BCG from a randomized phase 2 trial in patients with non-muscle invasive bladder cancer (NMIBC).
Date and Time: Sunday, June 4, 8:00 AM to 11:30 AM
Poster Number: 209

On May 30, 2017 XBiotech Inc. (NASDAQ:XBIT) reported an upcoming presentation of findings on key biomarker analysis of colorectal cancer patients treated with Hutruo (MABp1) in its European Phase III study (Press release, XBiotech, MAY 30, 2017, View Source [SID1234519319]).

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The abstract, entitled, "Pre-treatment Endogenous Interleukin-1 Receptor Antagonist (IL-1Ra) Levels in Metastatic Colorectal Cancer (mCRC) Patients are Associated with Clinical Outcomes After Anti-Interleukin-1a Therapy (MABp1)", will be presented by renowned oncologist, Dr. Razelle Kurzrock, Chief of Hematology & Oncology, UC San Diego School of Medicine. The data will be presented via poster presentation on Friday, June 30th from 10:30-11am and 4:40-5:10pm at the 19th ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer in Barcelona, Spain.

These findings indicate a significant association between pre-treatment circulating levels of IL-1Ra and responsiveness to MABp1 therapy. Lower pre-existing IL-1 antagonist activity was relatively responsive to pharmacological intervention with anti-IL-1α antibody therapy. This analysis provides new evidence that regulators of innate immunity may exert selection pressure on tumors and play an active role in the natural history of colorectal cancer. The impact of the body’s control of innate inflammation is thus found to affect the use of immune modulating therapy.

"Our analysis show a significant association between pre-treatment circulating levels of IL-1Ra and responsiveness to MABp1 therapy," stated Dr. Kurzrock. She further stated, "These results provide new insight on the active role for interleukin-1 regulation in disease progression in colorectal cancer and could help us create more personalized approaches to treatment of the disease."

About Razelle Kurzrock, M.D.
Dr. Kurzrock is a medical oncologist and a renowned expert in precision medicine. She is a thought leader in the use of anti-cytokine therapies for the treatment of cancer and one of the first to recognize the importance of the interleukin-1 pathway in cancer. While at the University of Texas MD Anderson Cancer Center, Dr. Kurzrock built one of the most successful Phase 1 clinical trials programs in the nation, and was the senior author in the pioneering study for the Company’s colorectal cancer study. Dr. Kurzrock currently serves as Senior Deputy Center Director for Clinical Science, Director at the Center for Personalized Cancer Therapy, Director of the Clinical Trials Office, and a Team Leader for Experimental Therapeutics at the Moores Cancer Center at UC San Diego. Dr. Kurzrock is also Chief of the Hematology & Oncology Division in the UC San Diego School of Medicine. Dr. Kurzrock serves on XBiotech’s Scientific Advisory Board.

About True Human Therapeutic Antibodies
XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.

On 30 May 2017 4SC AG (4SC, FSE Prime Standard: VSC) reported new preclinical data that demonstrates that resminostat influences the anti-cancer response of natural killer (NK) cells – a subset of our body’s own immune cells (Press release, 4SC, MAY 30, 2017, View Source [SID1234519317]). Resminostat increases the sensitivity of cancer cells to killing by NK cells and enhances the killing activity of NK cells towards cancer cells.

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Roland Baumgartner, Ph.D., Chief Scientific Officer of 4SC, explained the scientific details: "In earlier experiments we established that resminostat increased the sensitivity of cancer cells to NK cell-mediated killing. The addition of resminostat to numerous cancer cell lines induced expression of molecules such as NKG2D ligands – molecules that are specifically recognized by NK cells. In contrast to competing histone deacetylase (HDAC) inhibitors, which negatively affect the viability of NK cells, resminostat increased the proportion of NK cells in the tumor microenvironment.

We now demonstrated that treatment with resminostat enhanced the activity of NK cells. The addition of resminostat to either mixed blood cells or isolated NK cells resulted in strong activation of NK cells – measured by expression of the activation marker CD69. Furthermore, blood cells which were pre-treated with resminostat killed tumor cells more efficaciously than non-treated blood cells."

Jason Loveridge, Ph.D., Chief Executive Officer of 4SC, added: "These new preclinical data add another piece to our understanding of how resminostat affects the interplay between our body’s own immune system and cancer. These new insights are highly valuable and support our clinical development plans to advance resminostat to market authorization."



Oral presentation at the Cancer Immunotherapy and Combinations Congress

Svetlana Hamm, Ph.D., Head of Translational Pharmacology of 4SC, will present the scientific details at the Cancer Immunotherapy and Combinations Congress, as part of the World Preclinical Congress.
Presentation Epigenetic priming with HDAC inhibitor resminostat sensitizes cancer to NK cell based immunotherapy
Time Tuesday, 13 June 2017, 4:05 p.m. EDT
Location Westin Boston Waterfront, Boston, USA
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Further information

On May 30, 2017 Molecular Partners AG (SIX: MOLN), a clinical-stage biopharmaceutical company developing a new class of drugs known as DARPin therapies, reported that the first patient was dosed in the phase 2 multiple myeloma study of its lead oncology asset MP0250 (Press release, Molecular Partners, MAY 30, 2017, View Source [SID1234519316]). In the first phase 2 study, the efficacy and safety of MP0250 will be examined in combination with bortezomib (Velcade) and dexamethasone in patients with multiple myeloma who have failed standard therapies. The study will be performed in three different countries: Germany, Poland and Italy. Initial safety data are expected in 2017 and efficacy data in 2018.

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In addition, the company announced that MP0250 will also be further evaluated in solid tumors. Molecular Partners intends submit to the FDA in H2 2017 an Investigational New Drug Application (IND) for a phase 1b/2 trial of MP0250 in combination with osimertinib (Tagrisso) in EGFR-mutated T790M-positive Non-Small Cell Lung Cancer (NSCLC) patients. Osimertinib, a third-generation TKI targeting EGFR (Epidermal Growth Factor Receptor), has recently become the standard treatment for those NSCLC patients which harbor a T790M mutation. Despite this novel treatment, patients eventually relapse and treatments become ineffective. MP0250 offers the possibility to target two of the described escape pathways – HGF and VEGF. The combination of MP0250 with osimertinib is expected to continuously block the EGFR-mutated pathway and simultaneously inhibit two additional non-EGFR related pathways of resistance.

"We are pleased and proud to have reached this important milestone to dose the first patient in our multiple myeloma phase 2 study. This is the first step to test our hypothesis that MP0250 can address resistance pathways in various hematological and solid tumors. The submission of an IND for the first solid tumor indication will be another important milestone in the development program of our lead oncology asset MP0250 later this year," commented Dr. Andreas Harstrick, Chief Medical Officer at Molecular Partners.

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage in more than 5 targets at once, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics.

The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their good safety profile, low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapies have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology, and is advancing a proprietary pipeline of DARPin drug candidates in oncology. The most advanced global product candidate is abicipar, a molecule currently in Phase 3, in partnership with Allergan.

Several DARPin molecules for various ophthalmic indications are also in development. The most advanced systemic DARPin molecule, MP0250, is in Phase 1 clinical development for the treatment of solid tumors and has entered into Phase 2 development for hematological tumors. In addition, Molecular Partners intends to further evaluate MP0250 for solid tumors in a phase 1b/2 trial for EGFR-mutated T790M-positive NSCLC. MP0274, the second-most advanced DARPin drug candidate in oncology, has broad anti-HER activity; it inhibits HER1, HER2 and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. MP0274 is currently moving into Phase 1. Molecular Partners is also advancing a growing preclinical pipeline that features several immuno-oncological development programs. DARPin is a registered trademark owned by Molecular Partners AG.