On January 8, 2018 Molecular Partners AG (ticker: MOLN), a clinical-stage biopharmaceutical company developing a new class of drugs known as DARPin therapies*, reported initial safety and efficacy data from its ongoing phase 2 study of MP0250 in multiple myeloma (MM) (Press release, Molecular Partners, AUG 8, 2018, View Source [SID1234522938]).

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Eight patients have been treated in the first cohort of 8 mg/kg MP0250 and were included in the safety analysis set. No dose-limiting toxicities (DLTs) have been reported to date. Most frequent adverse events were Grade 1 and 2, except two Grade 3 adverse events (thrombocytopenia and transitory liver enzyme elevation) observed in two patients.

Of seven response evaluable patients receiving MP0250 in combination with bortezomib and dexamethasone, three patients showed partial response (PR) and one patient minimal response (MR). Responses were analyzed according to the International Myeloma Working Group (IMWG) criteria.

"We are very pleased with the good initial safety and tolerability data we observed in the first eight patients treated with MP0250 in combination with bortezomib and dexamethasone. Furthermore, we see promising responses within this first dose cohort. We look forward to evaluating additional patients at higher doses of MP0250 with bortezomib and dexamethasone to treat resistant multiple myeloma patients," commented Dr. Andreas Harstrick, Chief Medical Officer at Molecular Partners.

MP0250 is a proprietary DARPin drug candidate neutralizing VEGF and HGF and thus blocking key escape pathways and resistance. Increases in VEGF and HGF are associated with disease progression in multiple myeloma and have been linked to poor prognosis: they are known to be able to stimulate neovascularization, bone destruction, and myeloma proliferation, migration, and adhesion in the bone marrow. MP0250 shows activity in many preclinical tumor models including in multiple myeloma models in which it enhances the effects of bortezomib on inhibition of M protein production and bone lysis and reduces invasion of tumor cells. MP0250 has shown a favorable safety profile in a phase 1 clinical study in 45 patients in advanced solid tumors.

In the phase 2 MM study, the safety and efficacy of MP0250 is examined in combination with bortezomib (Velcade) and dexamethasone in patients with relapsed and refractory multiple myeloma (RRMM) who have failed standard therapies. The study is performed in Germany, Poland and Italy. A total of 40 patients are planned to be treated, 12 patients in the dose-escalation phase (Part 1) to establish a safe dose, and an additional 28 patients in the dose-expansion phase (Part 2) resulting in a total of 34 patients at the target dose. Additional safety and efficacy data are expected by the end of 2018.

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics that open an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage more than five targets at once, offering potential benefits over those provided 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 a clinical POC study in multiple myeloma. In addition, Molecular Partners will evaluate MP0250 for the treatment of solid tumors in a phase 1b/2 trial in patients with epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC). MP0274, the company’s second-most advanced DARPin drug candidate in oncology, has entered into phase 1 clinical development. With its broad anti-HER activity, MP0274 inhibits HER1-, HER2-, and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. 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 8, 2018 Sanofi and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will accelerate and expand investment for the clinical development of the PD-1 (programmed cell death protein 1) antibody cemiplimab in oncology and dupilumab in Type 2 allergic diseases (Press release, Sanofi Genzyme, JAN 8, 2018, View Source [SID1234522976]). Both of these breakthrough therapies have the potential to benefit a number of different patient populations and this strategic investment will enable the companies to evaluate cemiplimab and dupilumab in broad clinical development programs.

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Under the terms of the expansion, the investment in cemiplimab will be increased to $1.64 billion, an increase of approximately $1 billion over the initial 2015 agreement and Sanofi and Regeneron will continue to equally fund cemiplimab development. The companies will also continue their investment in other immuno-oncology programs under their existing Immuno-oncology Discovery Agreement. Investigational cemiplimab is being studied as monotherapy and in combination with other therapies in a wide range of cancers including advanced skin cancers, non-small cell lung cancer, cervical cancer and lymphomas, with more studies in other indications planned to begin in 2018. The companies expect to submit U.S. and EU regulatory applications for cemiplimab in advanced cutaneous squamous cell carcinoma in the first quarter of 2018.

The additional investment in the dupilumab development program will help accelerate planned new studies in chronic obstructive pulmonary disease, peanut allergy and grass allergy as well as in patients who have multiple allergic conditions. These areas are in addition to ongoing dupilumab clinical development in pediatric atopic dermatitis, pediatric asthma, eosinophilic esophagitis and nasal polyposis. Dupixent (dupilumab) is approved for the treatment of adults with moderate-to-severe atopic dermatitis in the U.S. and EU and a U.S. supplemental biologics license application was submitted for uncontrolled, persistent asthma for patients aged 12 and over in the fourth quarter of 2017.

The additional investment will also accelerate and expand development of REGN3500, an IL-33 antibody, with studies expected to be conducted in atopic dermatitis, asthma and chronic obstructive pulmonary disease. The increased funding for dupilumab and REGN3500 will be pursuant to the existing Antibody License and Collaboration Agreement between the companies.

"The ongoing collaboration between Sanofi and Regeneron underscores our commitment to partnering in the development of medicines to treat significant unmet medical needs," said Elias Zerhouni, MD, Global Head of R&D at Sanofi. "The expansion of these clinical programs for both cempilimab and dupilumab should enable us to quickly identify treatment opportunities in other disease areas."

Regeneron has agreed to grant a limited waiver of the "lock-up" in the Amended and Restated Investor Agreement between the companies, so that Sanofi may sell a small percentage of the Regeneron common stock it owns to fund a portion of the cemiplimab and dupilumab development expansion. This waiver will allow Sanofi to sell in private transactions to Regeneron up to an aggregate of 1.4 million shares of Regeneron common stock through the end of 2020, representing approximately 6 percent of the 23.9 million shares of Regeneron common stock Sanofi currently owns. As of October 20, 2017 there were 107.4 million shares of Regeneron capital stock outstanding. If Regeneron decides not to purchase the shares, Sanofi will be allowed to sell those shares on the open market, subject to certain volume and timing limitations. Further details on the updated agreements are available in Regeneron’s current report on Form 8-K filed today.

Cemiplimab and dupilumab were invented by Regeneron using the company’s proprietary VelocImmune technology that yields optimized fully-human antibodies. Other than the approved uses of Dupixent, cemiplimab, Dupilumab, and REGN3500 are under clinical investigation and their safety and efficacy have not been fully evaluated by any regulatory authority.

On January 8, 2018 Epizyme presented Company Slide Deck as of January 8, 2018 (Presentation, Epizyme, JAN 8, 2018, View Source [SID1234522982]).

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On January 8, 2018 TG Therapeutics, Inc. (NASDAQ:TGTX) reported that Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, will present at the 36th Annual J.P. Morgan Healthcare Conference, being held at the Westin St. Francis Hotel in San Francisco, CA (Press release, TG Therapeutics, JAN 8, 2018, View Source [SID1234523029]). The presentation is scheduled to take place on Thursday, January 11, 2018 at 7:30am PT. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com.

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On January 8, 2018 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that it has successfully manufactured the first SPEAR T-cells for a patient at its Navy Yard facility in Philadelphia (Press release, Adaptimmune, JAN 8, 2018, View Source;p=RssLanding&cat=news&id=2325396 [SID1234522941]). In addition, Adaptimmune announced an agreement with Cell and Gene Therapy Catapult for vector production in the UK, which will ensure vector supply for its ongoing and future clinical studies.

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"We are making great strides to becoming a fully integrated cell therapy company. Our Navy Yard facility is now fully operational producing SPEAR T-cells for patients. In addition, we have vector supply into 2019, and the initiation of our own vector manufacturing capability at the Catapult facility will extend vector supply further," said James Noble, Adaptimmune’s Chief Executive Officer. "We will continue to work with our cell manufacturing partner PCT, now part of Hitachi, where we have dedicated space and personnel for production of our SPEAR T-cells, as well as our other vector suppliers. Having these dedicated resources both in-house and through external partnerships is essential to ensure our future success as a fully integrated cell therapy company."

First SPEAR T-cells manufactured at the Navy Yard
The first SPEAR T-cells have been successfully manufactured by the Adaptimmune team at our own Navy Yard headquarters for a patient in the first dose cohort of the ongoing MAGE-A4 multiple tumor study in bladder, melanoma, head & neck, ovarian, non-small cell lung, esophageal, and gastric cancers.

The manufacturing facility at the Navy Yard can deliver cells for up to 300 patients per year, with the possibility of expansion that would enable manufacture for up to 1000 patients per year. In addition to production at its wholly-owned manufacturing facility at the Navy Yard, Adaptimmune will continue working with the PCT team to manufacture SPEAR T-cells.

Vector supply extended to beyond 2020
The agreement, which was executed on January 5, 2018 with Cell and Gene Therapy (CGT) Catapult, will enable Adaptimmune to have its own dedicated vector manufacturing space in the UK. It will ensure vector supply production beyond 2020 for ongoing studies with all three SPEAR T-cell therapies, MAGE-A4, MAGE-A10 and AFP.

The module, in which Adaptimmune will use its own novel vector manufacturing process and be responsible for operation of the manufacturing process, is located in the UK-based CGT Manufacturing Centre. The CGT manufacturing Centre is a Good Manufacturing Practice (GMP) facility designed to enable the development of commercial scale manufacturing systems in cell and gene therapy by offering a full suite of GMP facilities, support and expertise.