On September 6, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported that its CEO, Jan van de Winkel, Ph.D., will give a company update at the Morgan Stanley 16th Annual Global Healthcare Conference in New York, New York at 2:15 PM EDT / 8:15 PM CEST on September 12, 2018 (Press release, Genmab, SEPT 6, 2018, View Source [SID1234529323]). A webcast of the update, which will include brief opening remarks followed by a question-and-answer session, will be available on Genmab’s website at View Source

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On September 6, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, and SpringWorks Therapeutics, a clinical-stage rare disease and oncology company focused on sourcing and developing innovative treatments for underserved patient populations, reported that the companies have entered into a global clinical collaboration agreement to evaluate the safety, tolerability and preliminary efficacy of combining BeiGene’s investigational RAF dimer inhibitor, lifirafenib (BGB-283) and SpringWorks Therapeutics’ investigational MEK inhibitor, PD-0325901, in patients with advanced solid tumors (Press release, BeiGene, SEPT 6, 2018, View Source;p=irol-newsArticle&ID=2366390 [SID1234529325]). Under the terms of the agreement, BeiGene will be responsible for administering the Phase 1b clinical trial that is expected to commence during the first quarter of 2019 in patients with advanced solid tumors that harbor RAS, RAF mutations and other MAPK pathway aberrations, with all costs of the clinical studies and governance responsibilities to be shared equally among both parties. SpringWorks Therapeutics will also oversee fixed-dose formulation work as part of the collaboration.

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"BeiGene is committed to developing innovative medicines for cancer patients with little or no treatment options. We are delighted to work with SpringWorks Therapeutics to explore the potential benefits of this combination in patients with RAS mutations, a patient population with high unmet medical needs," said John V. Oyler, co-founder, chief executive officer and chairman of BeiGene.

"Mutations in RAS genes are found in roughly one-fourth of all human cancers, making this a critically important area for developing new cancer treatments. Despite decades of research, no anti-RAS therapies have been approved to date," said Saqib Islam, chief executive officer of SpringWorks Therapeutics. "The combination of a MEK inhibitor with a RAF dimer inhibitor has strong scientific rationale, and we look forward to partnering with BeiGene to build upon the existing preclinical data, which have demonstrated potential benefits with this combination therapy approach."

About Lifirafenib

Lifirafenib was discovered in BeiGene’s research facilities in Beijing, China, and is an investigational small molecule kinase inhibitor with RAF monomer and dimer inhibition activities. Lifirafenib has shown antitumor activities in preclinical models and in cancer patients with tumors harboring BRAF V600E mutations, non-V600E BRAF mutations, non-small cell lung cancer and endometrial cancer harboring KRAS mutations. To date, lifirafenib has been dosed in more than 150 patients globally.

About PD-0325901

PD-0325901 was originally discovered by Pfizer scientists and is an inhibitor of MEK, a key signaling protein for cellular survival and proliferation. PD-0325901 has been shown in clinical biopsies to block MEK phosphorylation, thereby arresting cellular growth and causing cell death to occur. Preclinical models have demonstrated significant synergy between MEK and RAF inhibition in RAS-mutant solid tumors. By vertically inhibiting the MAPK pathway, the combination approach of PD-0325901 and lifirafenib may potentially overcome feedback loops that have impeded therapeutic development for RAS-mutant solid tumors.

SpringWorks Therapeutics also plans to initiate a Phase 2b study of PD-0325901 as monotherapy for neurofibromatosis type 1 patients with plexiform neurofibromas in 2019. The company is also continually evaluating new licensing and partnership opportunities for its MEK program and is seeking other innovative partnerships designed to complement the existing clinical programs to advance its ambition of developing novel therapies for underserved patient populations.

On September 6, 2018 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmic company, reported the financial results for the Nicox Group for the six months ending June 30, 2018 and provided an update on its activities (Press release, NicOx, SEP 6, 2018, View Source [SID1234529343]).

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Michele Garufi, Chairman and Chief Executive Officer of Nicox, said: "Nicox is entering a new and exciting phase with the initiation of the Phase 2 study for our lead product candidate NCX 470 for IOP reduction in patients with open-angle glaucoma or ocular hypertension, and by strengthening our U.S. presence in our new site in Research Triangle Park in North Carolina. We have assembled the right team to achieve our important near term clinical and corporate milestones and continue delivering on all objectives in line with our growth strategy."

Key Upcoming Milestones
Q1 2019: Planned Investigational New Drug (IND) submission to the United States (U.S). Food and Drug Administration (FDA) for NCX 4251 to enable a Phase 2 clinical study in patients with acute exacerbations of blepharitis.
Q1 2019: Expected delivery of ZERVIATETM (cetirizine ophthalmic solution), 0.24% commercial product to Eyevance Pharmaceuticals LLC, followed by a launch for the spring 2019 allergy season in the U.S.
H2 2019: Expected top-line data from the NCX 470 Phase 2 study for the reduction of intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension.

Product and Product Candidates Updates
A Phase 2 study was initiated in Q3 2018 for Nicox’s product candidate NCX 470, a novel second generation nitric oxide (NO)-donating prostaglandin analog. This multicenter, double-masked, 28-day, parallel group, dose response study aims to evaluate the efficacy and safety of NCX 470 compared to latanoprost 0.005% in adult patients with elevated IOP due to open-angle glaucoma or ocular hypertension. The study is expected to randomize 420 patients in clinical sites across the U.S. The primary endpoint of the study is the mean reduction in diurnal IOP after 4 weeks of treatment, while the overall objective is to identify the appropriate dose of NCX 470 to be advanced into Phase 3 studies. This Phase 2 study was initiated following the submission of an IND application in June 2018, ahead of the previously disclosed target date of the third quarter of 2018. Nicox expects to report top-line data from this Phase 2 study in the second half of 2019.

Preclinical and formulation development of Nicox’s product candidate NCX 4251, a novel, patented ophthalmic suspension of fluticasone propionate nanocrystals, is continuing on track for the Q1 2019 IND submission to the U.S FDA to enable a Phase 2 study to evaluate the safety and efficacy of NCX 4251 compared to its vehicle in patients with acute exacerbations of blepharitis. An additional and positive pre-IND meeting was held with the U.S. FDA in June 2018, which addressed specific questions on development, including the potential primary endpoints. Based on FDA feedback, we are finalizing the design of the first-in-human Phase 2 study.

Two molecules from our future generation stand-alone NO-donors that target IOP reduction, NCX 667 and NCX 1660, are currently in formulation development and testing with the Re-Vana EyeLiefTM technology under the research collaboration agreement signed in October 2017. Depending on the release profile of these molecules with this technology in preclinical animal models of ocular pharmacokinetics, they may be advanced into further development and/or we may decide to test other molecules in the same technology.
Our research activities continue both in our research collaboration agreement with Ironwood, announced in June 2018, which is focused on combining Ironwood’s expertise in soluble guanylate cyclase with our proprietary NO-donating research platform, and in our internal programs combining NO with other undisclosed pharmacological mechanisms of action. We expect to be able to announce a preclinical candidate from one of these programs in the next 18 months.

VYZULTATM (latanoprostene bunod ophthalmic solution), 0.024% is now a revenue generating asset for Nicox, following the U.S. launch in December 2017 by partner Bausch + Lomb. In March 2018, the Company announced an amendment to the global licensing agreement under which royalties paid to Nicox on worldwide net sales of VYZULTA will increase by 1% over the original royalty on net sales above $300 million per year. In addition, the potential milestone payments payable to Nicox by Bausch + Lomb have been increased by $20 million.
We expect to ship commercial product and trade samples for ZERVIATETM (cetirizine ophthalmic solution), 0.24% to our partner Eyevance by Q1 2019, which will allow Eyevance to launch ZERVIATETM in the United States in time for the 2019 spring allergy season. The shipment of product triggers a $1 million milestone payment to Nicox by Eyevance, with up to $3 million of potential future milestones payments related to certain regulatory acceptance provisions and certain near term manufacturing objectives.
H1 2018 Financial Summary
Net revenue1 for the first half of 2018 was €0.3 million, comprised exclusively of royalties on H1 2018 sales of VYZULTATM by global partner Bausch + Lomb, after deduction of royalty payments due by Nicox. The Nicox Group recorded no revenues for the first half of 2017.

The operating expenses for the first half of 2018 were consistent with the same period last year (€10.0 million for the first six months of 2018 compared to €10.2 million for the first six months of 2017).

The Nicox Group recorded a net loss of €7.6 million for the six months ended June 30, 2018, compared to a net loss of €12.2 million for the same period in 2017.

As of June 30, 2018, the Nicox Group had cash and cash equivalents of €32.7 million as compared with €36.3 million at March 31, 2018 and €41.4 million at December 31, 2017.

Reference
1. Net revenue consists of revenue from collaborations less royalty payments which corresponds to Net profit from collaborations in the condensed consolidated statements of profit or loss for the six-month periods ended June 30, 2018.

The diligences related to the half-year review were performed by the auditors. The review report will be issued once procedures will be finalized over the half-year financial report.

On September 5, 2018 Innate Immunotherapeutics reported that it has elected to change the Company name to Amplia Therapeutics Limited and the Company stock code will change to ATX from IIL (Press release, Innate Immunotherapeutics, SEPT 5, 2018, View Source [SID1234529286]). The change received shareholder approval in a Special Resolution at the Company’s Annual General Meeting held on 30 August in Melbourne.

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The change of name is part of a wider branding refresh of the Company as it focuses on the development of a pipeline of Focal Adhesion Kinase (FAK) inhibitors for cancer and fibrosis. FAK is an important therapeutic target that is significantly upregulated in highly fibrotic tumours such as pancreatic and ovarian cancer. Drugs targeting FAK have the potential to sensitize the tumour microenvironment to both immuno-oncology drugs and chemotherapies.

Amplia Chairman Dr. Warwick Tong, noted "The Amplia name reflects the therapeutic action of inhibiting FAK to ‘amplify’ the effect of existing immuno-oncology and chemotherapeutic drugs in a number of difficult to treat cancers".

Amplia’s lead drug candidate AMP945 is expected to have a significant clinical advantage over other FAK-targeting molecules because of its high potency and selectivity for FAK. Amplia is also developing AMP886, which is a multi-action inhibitor that, in addition to potent FAK activity, also modulates FLT3 and VEGF, both highly synergistic targets in a number of solid and hematologic cancers. The development of these drug candidates is supported by an international team of worldclass reseachers and clinicians.

On September 5, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to LOXO-292, a selective RET inhibitor, for (Press release, Loxo Oncology, SEPT 5, 2018, View Source [SID1234529287]):

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the treatment of patients with metastatic RET-fusion-positive non-small cell lung cancer who require systemic therapy and have progressed following platinum-based chemotherapy and an anti-PD-1 or anti-PD-L1 therapy; and for
the treatment of patients with RET-mutant medullary thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options.
"We look forward to working with FDA to streamline the development of LOXO-292 in the two patient populations that have comprised the bulk of our initial clinical trial enrollment," said Josh Bilenker, M.D., chief executive officer at Loxo Oncology. "Given the many available therapies for non-small cell lung cancer and medullary thyroid cancer, we are pleased that LOXO-292 has shown encouraging data in refractory patients, and hope to demonstrate the full potential of this treatment in additional populations over time."

The LOXO-292 Breakthrough Therapy Designation was based on data from the ongoing global Phase 1/2 LIBRETTO-001 clinical trial. In 2019, the company plans to provide an update on the overall long-term LOXO-292 clinical development plan, based on feedback from global regulators.

The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of a drug candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

About LOXO-292
LOXO-292 is an oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. RET fusions and mutations occur across multiple tumor types with varying frequency. LOXO-292 was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms that could otherwise limit the activity of this therapeutic approach. LOXO-292 has been granted Breakthrough Therapy Designation by the U.S. FDA.

LOXO-292 is currently being studied in the global LIBRETTO-001 Phase 1/2 trial. For additional information about the LOXO-292 clinical trial, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient RET Clinical Trial Hotline at 1-855-RET-4-292 or email [email protected].

About RET-Altered Cancers
Genomic alterations in the RET kinase, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth. RET fusions have been identified in approximately 2% of non-small cell lung cancer, 10-20% of papillary and other thyroid cancers, and a subset of other cancers. Activating RET point mutations account for approximately 60% of medullary thyroid cancer (MTC). Both RET fusion cancers and RET-mutant MTC are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as "oncogene addiction," renders such tumors highly susceptible to small molecule inhibitors targeting RET.