On September 27, 2021 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX) and Incyte (Nasdaq: INCY) reported that they have entered into an exclusive worldwide collaboration and license agreement to develop and commercialize axatilimab, Syndax’s anti-CSF-1R monoclonal antibody (Press release, Incyte, SEP 27, 2021, View Source [SID1234590286]).

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"This partnership has the potential to significantly expand and maximize the axatilimab program across multiple lines of treatment in chronic graft-versus-host Disease (cGVHD), as well as additional indications in which the monocyte-macrophage lineage plays a vital role in the fibrotic disease process, such as idiopathic pulmonary fibrosis (IPF)," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Incyte is a proven leader in the development and commercialization of many important innovative therapies, including a treatment for GVHD. We are thrilled to be working alongside this talented and determined team to combine our expertise as we strive to provide new treatment options for patients in desperate need of effective interventions."

"We are excited to partner with Syndax and for the opportunity to bring another potential treatment to patients with life-threatening conditions, like GVHD," said Hervé Hoppenot, Chief Executive Officer of Incyte. "Collaborations between companies like Incyte and Syndax, who are both dedicated to scientific advancement, contribute to the development of new innovative medicines that may benefit patient communities around the world."

Syndax and Incyte are seeking to develop axatilimab as a backbone therapy for patients with cGVHD as well as in additional immune-mediated diseases where CSF-1R-dependent monocytes and macrophages are believed to contribute to organ fibrosis. Syndax recently completed a Phase 1/2 trial of axatilimab in patients with cGVHD. Data from the Phase 1 portion of the trial highlighting the tolerability and high response rate of axatilimab in cGVHD patients refractory to multiple therapeutic agents were reported during an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020. Updated results from the Phase 1 portion and preliminary results from the Phase 2 expansion portion of the study, which evaluated 1 mg/kg of axatilimab every two weeks, are expected to be presented at a medical meeting in the fourth quarter of 2021.

Enrollment continues in the ongoing global pivotal Phase 2 AGAVE-201 trial of axatilimab monotherapy in patients with cGVHD, with topline data expected in 2023. The companies also plan to initiate additional trials of axatilimab in patients with cGVHD in 2022, including a Phase 2 trial in combination with a JAK inhibitor in patients with steroid-refractory cGVHD. Beyond cGVHD, Syndax plans to commence a Phase 2 proof of concept trial of axatilimab early next year in patients with IPF, a serious, life-limiting orphan disease for which axatilimab could represent a much-needed treatment option with a novel mechanism of action.

Terms of the Collaboration

Under the terms of the agreement, Incyte will lead global commercial activities for axatilimab across all indications. The companies will participate in a 50:50 profit share in the U.S., and Syndax will receive double-digit royalties on sales outside of the U.S. Syndax will retain the option to co-promote axatilimab for any approved indications in the U.S. In connection with the agreement, Syndax will receive an upfront payment of $117 million plus a $35 million equity investment, which will be purchased at $24.62 per share, a 30% premium to the volume weighted average price over the 10 days prior to September 24, 2021. Syndax will also be eligible to receive up to an additional $450 million in potential regulatory, development and commercial milestone payments.

The companies will share development costs associated with global and U.S.-specific trials for all agreed upon trials at a rate of 55% (Incyte) and 45% (Syndax), with Incyte responsible for 100% of future development costs for trials that are specific to ex-U.S. countries. Syndax will fund the initial development of axatilimab in IPF and Incyte will have the option to co-fund late-stage development for this indication.

The agreement between Syndax and Incyte, including the upfront payment and equity investment, is subject to clearance by the U.S. antitrust authorities under the Hart-Scott-Rodino Act and will become effective as soon as these conditions have been met.

Goldman Sachs & Co. LLC is acting as the exclusive financial advisor to Syndax.

Syndax Conference Call and Webcast

In connection with this announcement, Syndax’s management team will host a conference call and live audio webcast at 8:00 a.m. ET today, September 27, 2021.

The live audio webcast may be accessed through the Events & Presentations page in the Investors section of Syndax’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 9875536
Domestic Dial-in Number: (855) 251-6663
International Dial-in Number: (281) 542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of Syndax’s website, www.syndax.com.

Incyte Conference Call and Webcast

Incyte will also host an analyst and investor conference call and webcast at 10:00 a.m. ET to discuss today’s news and the Company’s recent product approval in chronic GVHD. The live and archived webcast will be available via investor.incyte.com.

To access the conference call, please dial 877-407-3042 for domestic callers or +1-201-389-0864 for international callers (conference identification number 13723505).

If you are unable to participate, a replay will be available for 90 days. The replay dial-in number for the United States is 877-660-6853 and the dial-in number for international callers is +1-201-612-7415 (conference identification number 13723505).

About Chronic Graft-Versus-Host Disease

Chronic graft-versus-host disease (cGVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT) which can last for years. Chronic GVHD is estimated to develop in approximately 40% of transplant recipients, and affects approximately 14,000 patients in the U.S.1,2 Chronic GVHD typically manifests across multiple organ systems, with skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.3

About Idiopathic Pulmonary Fibrosis

Idiopathic Pulmonary Fibrosis (IPF) is a serious, life-limiting chronic lung disease characterized by fibrosis and scarring of lung tissue with a median survival of 3-5 years after diagnosis. Patients with IPF experience debilitating symptoms including progressive shortness of breath, particularly with exertion, chronic cough, fatigue, weakness, and chest discomfort. Currently approved drugs slow but do not halt disease progression and the only curative therapy is lung transplant, which is an option for less than 5% of patients. Estimates indicate that IPF could affect approximately 150,000 patients in the U.S. and approximately 260,000 patients across the seven major pharmaceutical markets (U.S., Japan, UK, Spain, Germany, Italy, and France).4

About Axatilimab

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases, such as chronic graft-versus-host disease (cGVHD) and idiopathic pulmonary fibrosis (IPF). Axatilimab data has demonstrated deep, durable responses and multiorgan clinical benefit in patients with cGVHD refractory to multiple therapeutic agents, and is currently being evaluated in the global pivotal Phase 2 AGAVE-201 trial in patients with cGVHD. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD and IPF. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.

On September 27, 2021 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery, reported that CEO Snehal Patel will present at the Benzinga Healthcare Small-Cap Conference being held September 29-30, 2021 (Press release, Greenwich LifeSciences, SEP 27, 2021, View Source [SID1234590285]).

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On September 29, 2021 at 9:30 am ET, Mr. Patel will present live to Benzinga’s small-cap investors. For more information and free registration, please visit the conference website at: View Source

Mr. Patel will also participate in a live interview on Benzinga’s Power Hour, which will occur at a later date.

About the Benzinga Healthcare Small Cap Conference

The Benzinga Healthcare Small Cap Conference connects small cap companies, investors, and traders. The conference includes live virtual company presentations, one-on-one meetings, and educational modules.

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 282,000 new breast cancer patients and 3.8 million breast cancer survivors in 2021. HER2/neu (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

On September 27, 2021 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported the financial results for Nicox SA and its subsidiaries (the "Nicox Group") for the six months ending June 30, 2021 and provided an update on recent events as well as progress on key programs (Press release, NicOx, SEP 27, 2021, View Source [SID1234590282]).

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First Half 2021 Financial Highlights
Net revenue1 for the first half of 2021 was €1.3 million (including €1.2 million in royalty revenue) versus €2.4 million (including €1.4 million in royalty revenue) for the first half of 2020. Operating expenses for the first half of 2021 were €13.3 million compared to €10.2 million for the first half of 2020.

The Nicox Group recorded a net loss of €11.7 million for the six months ended June 30, 2021, compared to a net loss of €14.6 million for the same period in 2020.
As of June 30, 2021, the Nicox Group had cash and cash equivalents of €36.5 million, as compared with €42.0 million at March 31, 2021 and €47.2 million at December 31, 2020. The Company is financed for at least 12 months, based on the development of NCX 470 alone.
As of June 30, 2021, the Nicox Group had financial debt of €18.0 million consisting of €16.0 million in the form of a bond financing agreement with Kreos Capital signed in January 2019 and a €2 million credit agreement guaranteed by the French State and granted in August 2020 in the context of the COVID-19 pandemic. Details of the bond financing agreement can be found in the Press Release of January 29, 2021.
Recent Events
The Mississippi Phase 2b clinical trial of NCX 4251 ophthalmic suspension 0.1%, evaluated against placebo, while not meeting the pre-defined blepharitis primary endpoint, demonstrated statistically significant results on blepharitis signs and symptoms when measured as change from baseline. NCX 4251 was found to be safe and well-tolerated over 14 days’ treatment, with no serious adverse events (see Press Release of September 23, 2021).
VYZULTA (latanoprostene bunod ophthalmic solution), 0.024%, recently received regulatory approval in Brazil, Jordan and Singapore. Further approvals and launches of VYZULTA will now be reported by our global partner Bausch + Lomb on a quarterly basis.
José Boyer has retired as Interim Head of R&D, although he will continue to advise the Company as a consultant. We would like to thank him for his commitment and contribution to Nicox.
Fera’s resubmitted application for Orphan Drug Designation (ODD) for naproxcinod in sickle-cell disease has been refused by the U.S. Food and Drug Administration (FDA), and Fera is currently seeking to discuss the next steps with the FDA. Fera continues to review other undisclosed therapeutic indications for naproxcinod.
Key Programs Updates
NCX 470: The New Drug Application submission timing to the U.S. FDA remains in line with our expectations, driven by the end of 2023 date for results from the ongoing Denali Phase 3 clinical In the first Phase 3 clinical trial, Mont Blanc, 494 out of 670 patients (74%) had been randomized as of September 20, and 398 of those patients have completed the 3-month efficacy evaluation. Given recruitment continues to be impacted by COVID-19, we are prudently moving the expectation for Mont Blanc trial top-line results to Q1 2023 (previously Q2 2022). The Phase 3 program, evaluating NCX 470 for lowering of intraocular pressure in patients with open-angle glaucoma or ocular hypertension, is expected to support NDA submissions in the U.S. and China, and will also provide data for countries accepting the same package for approval.
NCX 4251: Following the results of the Phase 2b Mississippi trial, the Company plans to meet with the U.S. FDA in early 2022 to discuss next steps in the development of this innovative product candidate for blepharitis.
ZERVIATETM in China: A Phase 3 clinical trial intended to support an application for regulatory approval in China, conducted and financed by our partner Ocumension, is ongoing.
Only figures at December 31, 2020 are audited, all other figures of this press release are non-audited.

Notes
Net revenue consists of revenue from collaborations less royalty payments which we refer to as net profit from collaborations in the condensed consolidated statements of profit or loss for the six-month period ended June 30, 2021.

On September 27, 2021 Astellas Pharma Inc. (TSE:4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and Seagen Inc. (Nasdaq:SGEN) reported that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved PADCEV (enfortumab vedotin) for radically unresectable urothelial carcinoma that has progressed after anti-cancer chemotherapy (Press release, Astellas, SEP 27, 2021, View Source [SID1234590281]). The New Drug Application received priority review.

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Radically unresectable urothelial carcinoma is urothelial cancer that cannot be treated by surgical removal of the urinary bladder or the kidney and the ureter due to tumor growth.

"Unfortunately, advanced urothelial cancer has a relatively poor prognosis and can be challenging to treat with currently available therapies," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Head of Development Therapeutic Areas, Astellas. "The MHLW’s review of enfortumab vedotin in just six months, supported by overall survival data from a pivotal Phase 3 clinical trial, reflects the seriousness of this condition and the potential benefit of enfortumab vedotin for patients in Japan."

The approval is primarily based on the global Phase 3 EV-301 clinical trial, which included sites in Japan. The trial evaluated enfortumab vedotin versus chemotherapy in adult patients with locally advanced or metastatic urothelial cancer who were previously treated with platinum-based chemotherapy and a PD-1/L1 inhibitor. At the time of pre-specified interim analysis, patients who received enfortumab vedotin (n=301) in the trial lived a median of 3.9 months longer than those who received chemotherapy (n=307). Median overall survival was 12.9 vs. 9.0 months, respectively [Hazard Ratio=0.70 (95% Confidence Interval [CI]: 0.56, 0.89), p=0.001]. The most common (≥20%) adverse reactions included alopecia, peripheral sensory neuropathy, pruritus, fatigue, decreased appetite, diarrhea, dysgeusia and nausea.

Each year in Japan, more than 24,300 people are diagnosed with bladder cancer and an estimated 9,500 die from the disease.1 Enfortumab vedotin is the subject of a robust clinical development program aimed at addressing unmet needs across the continuum of urothelial cancer and in other solid tumors.

On September 27, 2021 TAE Life Sciences (TLS), a biological-targeted radiation therapy company developing next-generation boron neutron capture therapy (BNCT), reported the successful installation of its neutron beam system at Xiamen Humanity Hospital, the most advanced accelerator-based BNCT facility in Greater China, newly built in partnership with Neuboron Medical Group (Press release, TAE Life Sciences, SEP 27, 2021, View Source [SID1234590272]).

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TLS’ Neutron Beam System enables a new treatment modality for patients diagnosed with the most aggressive and recurrent cancers such as gliomas, head and neck tumors, and melanomas, by delivering more precise, targeted radiation to cancer cells while sparing damage to surrounding healthy tissue. This technique holds promise in treating patients with cancer for whom other treatment options have been exhausted or are unavailable.

"The installation of our NBS at Xiamen Humanity Hospital represents a tremendous milestone in our mission to improve the lives of people diagnosed with invasive, recurrent and difficult to treat cancers," said Bruce Bauer, Chief Executive Officer of TAE Life Sciences. "TAE Life Sciences is also developing comprehensive boron target drugs and a neutron technology product portfolio to enable BNCT as a new cancer treatment modality for patients globally. With the global pandemic challenging our installation plans along with restrictions in travel, this milestone is even more meaningful, and we want to thank our colleagues at Xiamen Humanity Hospital and our partner in China, Neuboron, for all of their efforts in helping to make this possible."

Accelerating New Hope in the Fight Against Cancer: Presentations at ICNCT19

Committed to developing a new generation of targeted radiotherapy for patients with cancer, TAE Life Sciences will present 5 oral presentations at the19th International Congress on Neutron Capture Therapy (ICNCT19) on BNCT, occurring virtually September 27 – October 1, 2021.

Product and Facility Design Optimization for an Accelerator BNCT Building Expansion Project in Pavia, Italy
Development of new small molecule drugs for Boron Neutron Capture Therapy
Target selection and development of antibody boron conjugates for boron neutron capture therapy of head and neck cancer
Application of high fidelity radiation transport models to facility design, material selection, and shielding concepts for p-7Li accelerator based BNCT
Architecture, Implementation and First Performance Results of a Neutron Beam System for Accelerator BNCT
In addition, TLS’s partner for China, Neuboron, will be presenting an update on the design and installation of the BNCT facility at Xiamen Humanity Hospital.

For presentation details, visit: View Source

About Biologically Targeted Radiation Therapy (BNCT)

BNCT is a non-invasive, biologically targeted particle therapy that combines a non-toxic compound containing boron-10 and a low-energy, epithermal neutron beam. This combination generates a therapeutic dose at the cellular level that destroys tumor cells while sparing healthy tissues surrounding the tumor. This results in less toxicity for the patient and the potential to escalate the therapeutic dose to the cancer cells. BNCT has the potential to treat undetected metastases in the local region of the tumor in the same treatment session, which is difficult to impossible for conventional radiotherapy because of the logistical patient treatment setup barriers and the amount of radiation that would be delivered to healthy cells. In addition, BNCT may be utilized for several cancers which are resistant to many, if not all, current cancer therapies available, such as glioblastoma multiforme.