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.

On September 27, 2021 Amplia Therapeutics Limited (ASX: ATX) ("Amplia" or the "Company") reported that it will be providing a presentation at the Proactive Investors Lifesciences Webinar at 12:00 pm AEST on Tuesday 28 September 2021 (Press release, Amplia Therapeutics, SEP 27, 2021, View Source;[email protected] [SID1234590271]). The webinar will include results from the Company’s successful Phase 1 clinical trial of AMP945 and its plans for a Phase 2 clinical trial in first-line pancreatic cancer that is scheduled to commence in early 2022.

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To join the webinar, please register at: View Source

This ASX announcement was approved and authorised for release by the CEO of Amplia Therapeutics.

On September 26, 2021 JW Therapeutics (HKEx: 2126), an innovative biotechnology company focused on developing, manufacturing and commercializing cell immunotherapy products, reported updated 1-year follow-up result of relmacabtagene autoleucel injection (relma-cel) for the treatment of relapsed or refractory large B-cell lymphoma (r/r LBCL) at the 24th Annual Meeting of the Chinese Society of Clinical Oncology (CSCO) (Press release, JW Therapeutics, SEP 26, 2021, View Source [SID1234590275]).

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RELIANCE Study was a phase II, open-label, single-arm, multi-center clinical study in China, which included 59 adult patients with r/r LBCL who had failed at least 2 lines of previous treatment. As of the December 31st, 2020 data cut-off, in 58 evaluable patients, results include:

Relma-cel showed durable responses and long-term survival benefit; the Best Overall Response Rate was 77.6%, the Best Complete Response Rate was 51.7% and 1-year Overall Survival (OS) was 76.8% with a median follow-up of 17.9 months;
Relma-cel was generally well-tolerated with a safety profile including a low severe Cytokine Release Syndrome (≥grade 3) of 5.1% and a low severe neurotoxicity rate (≥grade 3) of 3.4%, and no new safety signals with a median of 17.9 months of follow up;
Long-term follow-up of the RELIANCE study have confirmed the durability of response and long-term OS with relma-cel treatment, which also was associated with low rates of CAR-T-associated toxicities.
"Large B-cell lymphoma (LBCL) is the most common and aggressive type of non-Hodgkin lymphoma and most patients relapse after initial therapies with limited treatment options with standard therapies and a median survival of approximately 6 months," said Mark J. Gilbert, Chief Medical Officer of JW Therapeutics, "These long-term follow-up data reported from the RELIANCE study have confirmed the durability of responses after relma-cel treatment, representing a potentially important treatment option for these patients that appears to offer clear hope of longer survival in these patients."

Reference:

Crump M, Neelapu SS, Farooq U, Van Den Neste E, Kuruvilla J, Westin J, Link BK, Hay A, Cerhan JR, Zhu L, Boussetta S, Feng L, Maurer MJ, Navale L, Wiezorek J, Go WY, Gisselbrecht C. Outcomes in refractory diffuse large B-cell lymphoma: results from the international SCHOLAR-1 study. Blood. 2017 Oct 19;130(16):1800-1808. doi: 10.1182/blood-2017-03-769620. Epub 2017 Aug 3. Erratum in: Blood. 2018 Feb 1;131(5):587-588. PMID: 28774879; PMCID: PMC5649550.

About Relmacabtagene Autoleucel Injection (trade name: Carteyva)

Relmacabtagene autoleucel injection (trade name: Carteyva) is an autologous anti-CD19 CAR-T cell immunotherapy products that was independently developed by JW Therapeutics based on a CAR T cell process platform of Juno Therapeutics (a Bristol Myers Squibb company). The first product of JW Therapeutics, relma-cel was approved by the China National Medical Products Administration (NMPA) in September 2021 for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, making it the first CAR-T product approved as a Category 1 biologics product in China. Currently, it is the only CAR-T product in China that has been simultaneously included in the National Significant New Drug Development Program, granted priority review and breakthrough therapy designations.

About RELIANCE Study (NCT04089215)

RELIANCE study was a single-arm, multi-center, pivotal study to evaluate the efficacy and safety of relmacabtagene autoleucel injection (relma-cel) in patients with relapsed or refractory large B-cell lymphoma (r/r LBCL) in China. At the time it was conducted, this study was the largest clinical study of CAR-T cell therapy in China under the Investigational New Drug (IND) pathway.

RELIANCE study enrolled 59 patients with r/r LBCL who had failed at least two lines of therapy, including a CD20 agent and anthracycline, and patients continue to be monitored for up to 2 years and beyond for long term outcomes. As of the December 31st, 2020 data cut-off, the Best Overall Response Rate was 77.6% with a Best Complete Response Rate of 51.7%, and 12-month Overall Survival (OS) was 76.8% in 58 evaluable patients; With a median follow-up of 17.9 months, of 59 treated patients, 5.1% and 3.4% of the patients experienced Cytokine Release Syndrome (CRS) and Neurotoxicity (NT) of Grade 3 or above, respectively. Rates for any severity grade CRS and NT were 47.5% and 20.3%, respectively. Long term follow up of the RELIANCE study have confirmed the durability of response and long-term OS with relma-cel treatment, which also was associated with low rates of CAR-T-associated toxicities.