On April 29, 2021 LIPAC Oncology LLC reported the conclusion of a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to discuss Phase 2b/3 clinical trials of LiPax in patients with low-grade highly recurrent non-muscle invasive bladder cancer (NMIBC) (Press release, Lipac Oncology, APR 29, 2021, View Source [SID1234578847]). The feedback from the FDA provides a clear path for LIPAC Oncology to initiate the Phase 2b trial for LiPax in the second half of 2021.

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"We appreciate the thoughtful feedback and guidance from the FDA and acceptance of our proposed design for the Phase 2b/3 trial of LiPax," said Michael Oefelein, M.D., Chief Medical Officer of LIPAC Oncology. "Non-muscle invasive bladder cancer is difficult to treat and highly recurrent. With the lack of existing treatment options for low to intermediate risk NMIBC, the unmet medical need is very high."

LIPAC Oncology recently announced the successful completion of a Phase 2a marker lesion clinical trial designed to predict long-term (two-year) recurrence free survival rates in patients with low-grade highly recurrent NMIBC treated with LiPax. In this study LiPax demonstrated a 63 percent marker lesion responder rate in highly recurrent and heavily pretreated patients. Based on these results, the FDA agreed that the Phase 2b trial will be a dose selection study with low dose (90 mg) and high dose (360 mg) arms and will enroll approximately 30 patients in each arm. The Phase 3 trial will be a randomized, double-blind, placebo-controlled trial in patients with low-grade recurrent NMIBC with a time to event primary endpoint after a one-year treatment phase.

"LIPAC Oncology is pleased to reach this important point in the development of LiPax, bringing us one step closer to providing this therapeutic option to the patients who could benefit from a novel approach to treatment," said Will Robberts, President of LIPAC Oncology. "We have made significant progress with the program, including executing a successful Phase 1/2a trial, securing a strategic partner in South Korea and expanding the global intellectual property protection with formulation patents being granted in Europe, Japan, China and other territories."

About NMIBC

Non-muscle invasive bladder cancer (NMIBC) is a cancer found in the tissue that lines the inner surface of the bladder. Bladder cancer is the sixth most common cancer in the U.S., and 74% of all bladder cancer is non-muscle invasive. Approximately 90,000 patients are newly diagnosed with NMIBC per year in the U.S. alone with as many as 390,000 patients per year newly diagnosed with NMIBC worldwide. The disease is marked by a high recurrence with a five-year recurrence rate averaging 50%.

Treatment of NMIBC is based on risk stratification. Patients are initially treated with a simple outpatient procedure involving transurethral resection of bladder tumor (TURBT) followed by a single, immediate instillation of intravesical chemotherapy to eliminate any residual tumor cells. Based on a patient’s biopsy results obtained from TURBT, NMIBC is stratified into three risk categories: low, intermediate, and high risk. To reduce recurrence and prevent progression, the American Urological Association NMIBC guidelines recommend induction and maintenance intravesical therapy after TURBT for intermediate risk patients. The low to intermediate risk category targeted by LiPax is estimated to comprise 95,000 Americans per year, yet no intravesical agent is approved by the FDA for this disease.

About LiPax

LiPax is a precision targeted, locally delivered taxane in Phase 2b development for intravesical instillation in the treatment of NMIBC. Its liposome-bound technology achieves targeted tissue penetration with no systemic exposure and toxicity. NMIBC is the lead program with additional orphan indications in upper tract urothelial cancer (UTUC), peritoneal and ovarian cancers and thoracic cancers (mesothelioma and malignant pleural effusion). LIPAC Oncology’s precision nano-technology platform provides targeted local liposomal delivery of taxanes (paclitaxel) to allow for deep tissue penetration in the bladder with no systemic exposure, dose-limiting toxicity or chemo-related side effects. LiPax is designed to enhance the standard of care of outpatient endoscopic tumor removal followed by intravesical instillation using a standard urinary catheter. LIPAC Oncology completed a Phase 2a clinical trial in August 2020 and intends to advance the program to a pivotal study to further investigate LiPax in the treatment of this condition.

On April 29, 2021 Sirnaomics Biopharmaceuticals (Suzhou) Co. Ltd., a subsidiary of Sirnaomics, Inc., a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer, fibrotic diseases and viral infections, reported that the company has entered into a partnership agreement with Walvax Biotechnology (Walvax) for the co-development of its anti-influenza siRNA therapeutic product candidate STP702 (Press release, Sirnaomics, APR 29, 2021, View Source [SID1234578846]). Sirnaomics will out-license to Walvax the exclusive development and commercialization rights for territories including mainland China, Taiwan, Hong Kong and Macao. Based on the agreement, the company will receive an initial payment of ~US$6.4 million for this asset, with additional milestone payments and royalty sharing based on product sales.

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While the medical and scientific communities are continuing the fight against the Covid-19 pandemic, there needs to be continued attention towards other viruses, such as Influenza, which cause severe epidemics worldwide. Along with its resistant strains, new pathogenic viruses continue to be discovered, creating an ongoing need for new anti-influenza treatments. RNA interference (RNAi) is a cellular gene-silencing phenomenon, in which sequence-specific degradation of target mRNA is achieved by means of complementary short interfering RNA (siRNA) molecules. This advanced therapeutic approach, using siRNA technology as a targeted inhibitor, affords a tractable strategy to combat influenza pathogenesis. SiRNAs are easy to design, and can be directed against multiple strains of the influenza virus by targeting their conserved gene regions. The newly established partnership between Sirnaomics and Walvax will specifically focus on development of the siRNA-based anti-influenza therapeutic candidate STP702.

"Establishing this partnership with Walvax exemplifies our continued execution for development strategy," said Patrick Lu, PhD, President and CEO of Sirnaomics, and Chairman of Sirnaomics Suzhou. "The out-licensing of our antiviral siRNA candidate, especially to an internationally well-recognized biopharma company like Walvax Bio that specializes in development of vaccine and therapeutics against various viral infections, marks a major milestone in Sirnaomics’ growth. I look forward to seeing the Sirnaomics and Walvax teams working together to develop a novel siRNA therapeutic to combat potential influenza epidemics of the future."

"We believe our siRNA technology has extremely broad therapeutic potential and we are excited to combine Walvax’s deep vaccine expertise with our siRNA platform technology to unlock additional value outside of our core oncology and fibrosis therapeutic focus," said Allan Shaw, Chief Financial Officer at Sirnaomics. "This alliance provides the latest validation of Sirnaomics technology platform and furthers our strategy to evaluate its potential in a wide range of new disease areas."

About STP702
Sirnaomics team has been working diligently on finding a better alternative for prophylaxis and therapeutic treatment of Influenza A. In silico design and in vitro screening (H1N1 in a P2- and H7N9 in a P3-environment) led to the identification of potent siRNA sequences targeting the most conserved regions of Influenza viral genes, and these siRNAs exhibited a broad anti-influenza activity. Through rational pairing of these potent siRNA oligos, a specific combination of two siRNA sequences can further improve efficacy by demonstrating synergistic antiviral activity while also broadening the potential coverage of Influenza strains. The most critical technical advancements are the nanoparticle-enhanced siRNA delivery (STP702) in the viral-challenged mouse models, resulting in more potent anti-influenza activity than the marketed chemo drugs: Ribavirin and Tamiflu.

These results are the first demonstration of a synergistic therapeutic effect from combining siRNAs in a single delivery system. They also demonstrate the utility of nanoparticle mediated delivery of siRNAs through IP administration and suggest that these siRNAs may act as a broad anti-Influenza therapeutic – offering a rapid response to newly emerging viral outbreaks where existing therapeutics and vaccines are ineffective. Finally, mutations in select gene segments within the virus can confer resistance to existing therapeutics, while the use of STP702 containing siRNAs against two distinct gene segments within the influenza viruses should greatly reduce the ability of the virus to escape therapeutic pressure and reduce this additional threat.

On April 29, 2021 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported that it will host a conference call and webcast on Wednesday, May 12, 2021 at 5:00 p.m. Eastern Time to review its first quarter 2021 financial and operating results and provide a corporate update (Press release, Marker Therapeutics, APR 29, 2021, View Source [SID1234578845]).

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The webcast will be accessible in the Investors section of the Company’s website at markertherapeutics.com. Individuals can participate in the conference call by dialing 877-407-8913 (domestic) or 201-689-8201 (international) and referring to the "Marker Therapeutics First Quarter 2021 Earnings Call."

The archived webcast will be available for replay on the Marker website following the event.

On April 29, 2021 Illumina, Inc. (NASDAQ: ILMN) reported that its executives will be speaking at the following investor conference (Press release, Illumina, APR 29, 2021, View Source [SID1234578844]):

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Bank of America 2021 Healthcare Conference on May 13, 2021
Fireside Chat at 11:45 am pacific time / 2:45 pm eastern time
The live webcast can be accessed under the Investor Info section of the "company" tab at www.illumina.com. Replays will be posted on Illumina’s website as soon as possible after the event and will be available for at least 30 days following.

On April 29, 2021 NANOBIOTIX (Paris:NANO) (NASDAQ:NBTX) (Euronext : NANO – NASDAQ: NBTX – the ‘‘Company’’), a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported operational progress and cash position for the first quarter of 2021 (Press release, Nanobiotix, APR 29, 2021, View Source [SID1234578843]).

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"The first quarter of this year saw further progress in development for Nanobiotix," said Laurent Levy, co-founder and chief executive officer of Nanobiotix. "We continue to generate data reinforcing our vision for NBTXR3 as a first-in-class radioenhancer that could have a significant impact for patients across solid tumor types and therapeutic combinations. We are also encouraged that Sanofi has seen the potential of Curadigm’s Nanoprimer and look forward to a successful collaboration between the two companies in gene therapy."

Financial Updates Supporting Nanobiotix Development and Corporate Plans

Cash, cash equivalents, and short-term investments were €107.1 million at March 31, 2021, supporting robust development plans into the second quarter of 2023, compared to €28m as of March 31st, 2020..

Following the decision to conclude a License and Collaboration agreement with PharmaEngine, Inc., the Company did not generate any revenue during the first quarter of 2021. This compares to €24k for the first quarter of 2020, which resulted from cross-charges associated with the collaboration.

Clinical Activities and Achievements Advancing Solid Tumor-Agnostic and Combination-Agnostic Potential of NBTXR3

Preclinical data, developed in collaboration with the University of Texas MD Anderson Cancer Center (MD Anderson), further suggesting that NBTXR3 could prime adaptive immune response and combine with several immune checkpoint inhibitors was presented at the first American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Special Conference on Radiation Science and Medicine in March. This data demonstrated that a combo therapy including NBTXR3, anti-PD-1, anti-TIGIT, and anti-LAG3 augmented anti-tumor response in both irradiated and unirradiated tumors, improving local and distant tumor control and increasing survival rate. The survivor mice were immune to re-injections of tumor cells, maintained significantly higher percentages of memory immune cells and stronger anti-tumor immune activities than control.
First clinical results in rectal cancer including recommended phase II dose from the complete phase Ib part of a phase Ib/II study evaluating NBTXR3 activated by radiation therapy with concurrent chemotherapy were presented in January at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO-GI 2021). The data showed that the intra-tumoral injection of NBTXR3 was feasible and well tolerated at all dose levels. More than 70% of patients showed objective tumor response and approximately 90% of patients underwent total mesorectal excision (surgery), and 17.6% achieved pathological complete response. In addition, 50% of the patients receiving surgery had good tumor regression. PharmaEngine, Inc. will implement the termination and wind-down of the phase II part of this clinical study pursuant to the agreement regarding the conclusion of this collaboration.
First patient injected in esophageal cancer, a new indication for NBTXR3, in a phase I study evaluating NBTXR3 activated by radiation therapy with concurrent chemotherapy (Study 2020-0122) in January. The trial is being conducted with MD Anderson as part of an ongoing clinical collaboration.
Corporate Activities and Achievements Expanding Value Creation Opportunities for Nanobiotix

Collaboration agreement between Sanofi and Nanobiotix subsidiary, Curadigm, in January. Pursuant to Sanofi’s selection of a project involving Curadigm’s Nanoprimer technology as a promising option to significantly improve gene therapy development, Curadigm entered into a one-year agreement with the pharmaceutical company inclusive of direct funding and scientific exchanges. The goal of the project is to establish proof-of concept for the Nanoprimer as a combination product that could improve treatment outcomes for gene therapy product candidates.
Retention of intellectual property rights for NBTXR3 in the Asia-Pacific region in March 2021 pursuant to a mutual agreement with PharmaEngine, Inc. to conclude the collaboration entered into in August 2012.
Upcoming Corporate and Clinical Events in the Second Quarter of 2021

May 24th: UBS Global Healthcare conference
June 1st: Jefferies Healthcare Conference
June 4th: American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) congress 2021
June 11th: Virtual KOL Event Review Potential Therapeutic Potential of NBTXR3 in Combination with Check Point Inhibitors for Patients with Advanced Cancers
Updated Financial Agenda

July 20th: Second Quarter Corporate Update and Financial Update
September 8: Half Year Corporate Update and Financial Update
October 20th: Third Quarter Corporate Update and Financial Update
About NBTXR3

NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

NBTXR3 is being evaluated primarily in locally advanced head and neck squamous cell carcinoma (HNSCC). The company-sponsored phase I dose escalation and dose expansion study has produced consistently favorable safety data and early signs of efficacy; and a phase III global registration is planned to launch in 2021. In February 2020, the United States Food and Drug Administration granted the regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the planned phase III.

Nanobiotix has also prioritized a company-sponsored Immuno-Oncology development program—a phase I clinical study evaluating NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors for patients with locoregional recurrent or recurrent/metastatic HNSCC and lung or liver metastases from any primary cancer eligible for anti-PD-1 therapy.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a strategic collaboration with strategy with world class partners to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 The University of Texas MD Anderson Cancer Center engaged in a broad, comprehensive clinical research collaboration with Nanobiotix to sponsor several phase I and phase II studies to evaluate NBTXR3 across tumor types and therapeutic combinations.