On January 3, 2018 Molecular Partners AG (SIX: MOLN), a clinical-stage biopharmaceutical company developing a new class of drugs known as DARPin therapies, reported that Allergan has exercised two options to develop and commercialize DARPin product candidates from its 2012 discovery alliance agreement with Molecular Partners (Press release, Molecular Partners, JAN 3, 2018, View Source [SID1234522825]). Upon receipt of approval under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, Molecular Partners will grant Allergan an exclusive license to the selected DARPin molecules for use in ophthalmology.

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Molecular Partners and Allergan entered into a broad discovery alliance in ophthalmology in 2012 aiming to develop novel multi-DARPin molecules for diseases with high unmet medical need. This alliance broadened the initial collaboration on abicipar, which is now in phase 3 development in wet AMD.

All amounts payable under these two option exercises are included in the aggregate milestone payments and the tiered royalty payments previously disclosed in our July 21, 2015 press release. Molecular Partners is entitled to certain success based development, regulatory and sales milestone payments aggregating up to USD 640 million, as well as tiered royalty payments (up to low double digit percentage range) on any future product sales. Allergan will be responsible for all future development costs.

"The DARPin platform is a key part of our strategy to develop highly differentiated drugs in ophthalmology," said David Nicholson, Chief R&D Officer, Allergan. "Our partnership with Molecular Partners continues to deliver such differentiated drug candidates and by exercising these options we will obtain exclusive rights to develop and commercialize these molecules and expand our efforts to address important diseases in ophthalmology. "

"We are excited to support our long-standing partner Allergan in advancing multi-DARPin product candidates. This is an important showcase of the value of the DARPin platform to deliver potential patient benefit in ophthalmology in addition to the work Molecular Partners is doing in oncology. I would like to thank the teams on both sides of the collaboration for their efforts and the achievement of this important milestone," commented Patrick Amstutz, CEO of Molecular Partners.

About abicipar
Abicipar is a long-acting mono-DARPin drug candidate that inhibits vascular endothelial growth factor A (VEGF-A) and is currently under investigation for the treatment of two major causes of blindness worldwide:
neovascular, or wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). Abicipar has the potential to require less frequent injections into the eye than the current anti-VEGF standards of care, while providing equal or better improvements in vision, both seen as mayor patient benefits in these indications. Molecular Partners granted an exclusive license to Allergan for Abicipar in May 2011.

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 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 has just moved 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.

On January 3, 2018 Elstar Therapeutics, a company fulfilling the promise of precision cancer immunotherapy through a powerful new approach to generating multi-functional therapies, reported the closing of a $39M Series A financing round by founding investor Apple Tree Partners (Press release, Elstar Therapeutics, JAN 3, 2018, View Source [SID1234522877]). The funds will be used to expand its growing pipeline and progress several early stage molecules towards the clinic.

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Elstar’s proprietary Universal Targeted Immunotherapy (UniTITM) platform allows rapid generation of multi-functional antibody-based molecules that enable patients to harness their own immune system to fight cancer. UniTITM molecules are unique in their composition of up to four distinct functional domains designed to regulate a broad-range of immunological activities, bringing unmatched targeting of tumor cells to reduce systemic toxicity while delivering excellent manufacturing and drug-like properties.

"Our multi-modal technology enables an enormous step forward from current generation cancer immunotherapy programs," said Steve Arkinstall, D.Phil., President and Chief Executive Officer of Elstar Therapeutics. "This financing will enable our experienced team to expand and advance our robust pipeline of cancer immunotherapy programs. Elstar’s goal is to rapidly move drug candidates into clinical trials for several serious hematological and solid malignancies."

"Elstar’s technology platform is designed to overcome barriers that have historically limited the success of promising immunotherapeutic approaches," said Elstar’s founder and Chief Scientific Officer Andreas Loew, Ph.D. "We are developing a new generation of drugs that can precisely target diseased cells. At the same time, these drugs can engage and regulate cells of both the adaptive and the innate immune system. While these molecules bring many biological advantages, at the core of the technology is the delivery of high yield molecules with drug-like properties that are manufactured using conventional antibody production and purification processes."

Elstar has assembled an accomplished leadership team to power its mission of being a part of the cure for cancer. The Company was founded by veteran protein engineer Andreas Loew, Ph.D., together with Apple Tree Partners, a life sciences-focused venture capital firm based in New York City. Before founding Elstar, Dr. Loew served as Executive Director of New Technologies / NIBR Biologics at the Novartis Institute for Biomedical Research, where he was responsible for the design and discovery of biotherapeutics including multi-specific antibodies and chimeric antigen receptor T cell therapies (CAR-T). Previously, Dr. Loew served in positions of increasing responsibility at Abbott Bioresearch Center.

Steve Arkinstall, D.Phil., Elstar’s President and Chief Executive Officer, has more than 25 years of experience in biopharmaceutical drug discovery research. Previously, he served in various roles at EMD Serono including President of the Serono Research Institute and SVP of Global Technologies before serving as Chief Scientific Officer of Kymab.

John Herrmann, Ph.D., serves as Chief Strategy Officer and EVP, Oncology. Dr. Herrmann is a portfolio strategist who previously was CSO of Oncology External Innovation at Eli Lilly & Co., before serving as SVP of Oncology at Catenion GmbH.

Peter Williams brings over twenty years of business development, deal negotiation and technology in-and out-licensing experience to Elstar as its Chief Business Officer. He has considerable expertise in technology and product partnering, particularly in the oncology landscape, having previously served as Senior Director, Business Development at Millennium Pharmaceuticals, Inc., Senior Director, Business Development at Alnylam Pharmaceuticals, Inc., and most recently as Vice President of Business Development at ImmunoGen, Inc.

"Elstar’s powerful UniTITM platform redefines how we think about multi-functional therapeutics and, together with a proven leadership team, positions the company to make a major impact on patients’ lives. Elstar’s initial focus on oncology has already generated candidates with great promise for various indications, but there are many other diseases where this technology could really transform the standard of care." said Aaron Kantoff, Principal at Apple Tree Partners and member of the Elstar Board of Directors.

On January 3, 2018 GeneCentric Therapeutics, Inc. reported a research collaboration to evaluate the potential of GeneCentric’s Cancer Subtype Platform (CSP) to identify responders to G1 Therapeutics’ oral CDK4/6 inhibitor G1T38 for the treatment of non-small cell lung cancer (NSCLC) (Press release, GeneCentric Therapeutics, JAN 3, 2018, View Source [SID1234522885]). As part of the research collaboration, GeneCentric will apply its lung cancer subtyping profilers (LSP) in preclinical, patient-derived xenograft models to determine NSCLC subtype associations with G1T38 response, as well as examine potential drug response associations with specific genes. Financial terms were not disclosed.

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GeneCentric’s proprietary core technology, CSP, identifies biologic subtypes of cancer through an integrated analysis of tumor genomics. The subtypes, which combine gene expression data with disease-related molecular pathways and immune cell expression have potential to function as universal biomarkers of drug response and enable more precision drug development. GeneCentric’s Lung Cancer Profilers (LSP 210 and LSP 220) define molecular subtypes of the two most common forms of NSCLC, adenocarcinoma and squamous cell carcinoma. Published studies have demonstrated that the subtypes, while indistinguishable by standard morphology, have different genetic, molecular and clinical attributes, and immune profiles that may drive drug response.

"G1T38 is a novel and promising targeted therapeutic with potential in many cancers," said Myla Lai-Goldman, M.D., CEO of GeneCentric. "This collaboration could identify subtypes of lung cancer where the compound is most likely to be efficacious."

On January 3, 2018 Asana BioSciences, an oncology-focused, clinical stage biopharmaceutical company, reported that the U.S. FDA has accepted the IND application for ASN007, a potent and selective ERK1/2 inhibitor (Press release, Asana BioSciences, JAN 3, 2018, View Source [SID1234522875]).

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"We are extremely pleased with the acceptance of this IND for evaluation of our potential Best-in-Class, oral ERK1/2 inhibitor that shows potent anti-proliferative activity in cancer models driven by MAP Kinase pathway mutations, as well as those resistant to BRAF and MEK inhibitors," said Sandeep Gupta, PhD, Founder, President and Chief Executive Officer at Asana BioSciences. "We continue to execute on our strategy of developing novel drugs that work on clinically validated targets, are clearly differentiated from the competition, and expected to offer significant benefit over the existing standards of care. This represents the 5th successful IND of our proprietary portfolio in the past 3 years, and is a testament to the capabilities, dedication and high efficiency of Asana’s R&D team," said Dr. Gupta.

The RAS/RAF/MEK/ERK (MAP Kinase) signaling pathway is frequently hyperactivated in a wide range of cancers through mutations in upstream targets such as BRAF, RAS and receptor tyrosine kinases. Inhibition of ERK1/2 offers a promising therapeutic strategy for these cancers, particularly those driven by RAS mutations. Enrollment in this Phase 1, open-label, dose-finding study of ASN007 in patients with advanced solid tumors is expected to start soon. The study will evaluate the safety, tolerability and preliminary efficacy of ASN007 in patients with BRAFV600, KRAS, HRAS or NRAS mutations.

On January 3, 2018 Rasna Therapeutics, Inc. (OTCQX: RASP), a clinical stage biotechnology company focused on the development of disease-modifying drugs for hematological malignancies, reported follow up Phase II clinical data showing that 4 out of 9 (44%) evaluable AML patients carrying the NPM1 gene mutation treated with actinomycin D ("Act D"), achieved complete remission (CR) (Press release, Rasna Therapeutics, JAN 3, 2018, View Source [SID1234522873]). Treatment with Act D was well tolerated, except that patients experienced oral mucositis as the major toxicity. Rasna Therapeutics has developed a proprietary nanoparticle based formulation of Act D (RASP-101), which is anticipated to maximize efficacy while minimizing oral mucositis. RASP-101 could potentially be a first-in-class modality for treatment of NPM1-mutated acute myeloid leukemia (AML).

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NPM1-mutated AML is a specific genetic leukemia entity that accounts for approximately one-third cases of AML in adults. As we reported previously, intravenous treatment of refractory or relapsed NPM1-mutated AML patients with Act D (12.5 µg or 15 µg/day) for 5 consecutive days produced hematological complete response in some of them (Falini et al., N Eng J Med. 373: 12, 2015).

In a follow-up phase II clinical study (ActD-AML-PG01, EudraCT 2014-000693-18) in refractory/relapse (R/R) AML patients carrying NPM1 gene mutation, treatment with Act D at 15 µg/kg/day for 5 days every 28 days induced CR in 4 out of the 9 evaluable patients (44.4%) with only 1 or 2 cycles of therapy. Three out of the 4 (75%) patients who obtained CR relapsed after 3, 5 and 7 months, respectively. One patient underwent haploidentical allogeneic PBSC transplantation at 3 months after CR achievement and is alive in molecular CR (MRD-negative) after 24 months.

"The precise mechanism of action of Act D in NPM1-mutated AML is still not clearly understood. Follow up data confirms our earlier findings and further support the use of Act D to induce complete hematological remissions with possible long-term molecular responses in NPM1-mutated AML patients. To note, our first previously reported NPM1-mutated AML patient (resistant to hypomethylating therapy) treated with Act D remains in molecular remission at over 3 years since CR achievement," said Dr. Brunangelo Falini, a member of the scientific advisory board of Rasna Therapeutics, Inc.

"We have developed a proprietary formulated Act D (RASP-101), which we anticipate will produce a superior clinical outcome with improved efficacy and safety profile. Emerging data from the multicenter clinical studies will allow us to develop a biomarker strategy to select responsive patients and improve clinical outcome for this unmet clinical need" commented Alessandro Padova, Chairman of Rasna.

About Actinomycin D (Dactinomycin)

Actinomycin D, also known as dactinomycin, a cytotoxic antibiotic produced by Streptomyces parvullus, was approved for medical use in the United States in 1964. It is on the World Health Organization’s List of Essential Medicines, the most effective and safe medicines needed in a health system. It is believed to work by blocking RNA synthesis. The drug has been used to treat a number of types of cancers, including Wilms tumor, rhabdomyosarcoma, Ewing’s sarcoma, trophoblastic neoplasm, testicular cancer, and certain types of ovarian cancer.

About Dr. Brunangelo Falini, M.D.

Brunangelo Falini is the head of the Institute of Hematology and Hemopoietic Stem Cell Transplantation at the University of Perugia, Perugia, Italy. His research activity has mainly focused on the genetic characterization of lymphomas and leukemias using monoclonal antibodies and, more recently, NGS technologies. He led the research group who discovered NPM1 mutations in AML in 2005 and the BRAF-V600E mutation in hairy cell leukemia in 2011. Both these seminal discoveries have already translated into a better diagnosis and therapy of patients affected by these hematological malignancies. Dr. Falini is the recipient of numerous prestigious prizes, including the "Josè Carreras Award" from EHA (Free EHA Whitepaper) (Barcelona, 2010), the "Leopold Griffuel Prize" from ARC (Paris, 2015) and the "Prize for Excellence in Medicine" from the American-Italian Cancer Foundation (New York, 2017).