On September 24, 2020 Nuformix plc (LSE:NFX) ("Nuformix" or "the Group"), a pharmaceutical development company focused on unlocking the therapeutic potential and value of known drugs to develop novel medicines to provide enhanced benefit, reported it has signed an exclusive option agreement with Oxilio Ltd ("Oxilio") (Press release, Nuformix, SEP 24, 2020, View Source [SID1234621605]).

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.Agreement covers a licence for the development and exploitation of NXP001 in oncology
· Up-front payment for an exclusive option period of 6 months, within which the global licensing agreement can be triggered
· Should Oxilio exercise the option, Nuformix will licence its patent estate and know-how on NXP001 in return for a significant upfront payment and additional development milestones and a royalty on net sales, capped at £2 million per annum

Under the terms of the agreement Nuformix will also provide a fixed amount of consultancy services to Oxilio during the option period. Oxilio will develop and seek to exploit NXP001 globally for the treatment of cancer and early clinical trials will determine which cancer types respond best to treatment. Cancer is the second leading cause of death globally with around 10 million deaths per annum, in spite of extensive studies to find new treatment regimens and more effective drugs. However, the cost of cancer treatment is increasing, in large part due to the expense of taking drugs through clinical trials where historically there is a very low rate of success. Therefore, there is an urgent need to develop safe, effective, and readily available anticancer agents.

Oxilio is focused on developing a therapeutic with the potential to treat patients with various cancer types representing a global therapeutics market estimated to be more than $130 billion (BCC Publishing, Jan 2019). Oxilio is focused on alleviating the current dilemma of a shortage of drug candidates for finding new cancer therapies, by adopting a drug repurposing strategy (identifying new uses for approved or investigational drugs that are outside the scope of the original medical indication). The major advantage of this approach is that the pharmacokinetics, pharmacodynamics and toxicity profiles of these drugs are already reasonably well established.

Thus, drug repurposing may hold the potential to result in a less risky development route with substantially lower associated development costs. The collaboration with Nuformix allows Oxilio to focus on developing rapidly a unique formulation and dosage form with NXP001. This would potentially overcome the key hurdle in drug repurposing, patent consideration-induced market exclusivity, as well as providing an accelerated route to the clinic.

Dr Chris Blackwell, Executive Chairman, said: "This agreement allows Nuformix the opportunity to benefit from the upside of a significant global market opportunity whilst realising short-term revenues. Furthermore, it demonstrates our commitment to explore and leverage value creating opportunities for all our pipeline assets. We look forward to working with Oxilio on this collaboration."

Dr Simon Yaxley, Co-Founder and Director of Oxilio said: "This collaboration with Nuformix allows Oxilio to continue the development of NXP001 as a potential new treatment in the battle against cancer. We are excited by the opportunity of accelerating our science towards our first clinical trials through this collaboration and we look forward to working with the Nuformix team to realise the opportunity".

About NXP001-Oncology
NXP001 has been developed by Nuformix, to date, with the aim of improving its use in the cancer critical care setting, particularly with regard to chemotherapy-induced nausea and vomiting (CINV). Business development activities continue with this focus alongside the option agreement for Oxilio to explore its potential in treating cancers.

About Cocrystals Pharmaceutical cocrystals are materials composed of two or more different molecules, usually an active pharmaceutical ingredient together with a pharmaceutically acceptable "coformer" molecule. Cocrystals can be engineered to enhance the bioavailability, pharmacokinetics, stability and manufacturing of drug products.

On September 24, 2020 UbiVac, www.ubivac.com, a private, clinical-stage immuno-oncology company with a lead product that educates the immune system to recognize and destroy cancer, reported that its co-founder and CEO, Bernard A. Fox, PhD, will speak at the Chinese Society for Clinical Oncology Conference held 19-26 September in Xiamen, China (Press release, UbiVac, SEP 24, 2020, View Source [SID1234570404]).

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During his remarks, Dr. Fox will outline findings of biomarker studies documenting that patients lacking anti-cancer immunity will respond significantly less well to standard cancer therapies or checkpoint blockers. These studies suggest that to improve patient outcomes, it will be necessary to induce or boost anti-cancer immune responses.

Dr. Fox will then review preclinical and clinical data that led to the development of a first-in-human triplet cancer immunotherapy trial. This multicenter randomized clinical trial includes: 1) a cancer vaccine, DPV-001, known to activate the anti-cancer immune response to a spectrum of cancer antigens, 2) a T cell agonist, anti-OX40, that can boost anti-cancer immune responses, followed by 3) delayed administration of anti-PD-1, to relieve checkpoint inhibition.

The cancer vaccine, UbiVac’s DRibble immunotherapy (DPV-001) is a first-in-class technology that combines more than 100 cancer antigens overexpressed by triple negative breast cancer (TNBC) with multiple immune stimulants in microvesicles that are targeted to dendritic cells. "DRibble vaccines are a disruptive technology documented to induce broad anti-cancer immune responses in mice and humans," said Dr. Hong-Ming Hu, UbiVac co-founder and CSO. He continued, "however, our preclinical studies suggest that to increase the curative potential you need to add a booster, that is where anti-OX40 comes in". Dr. Hu’s previous work, published in Scientific Reports in 2016, identified substantial and significant increases in long-term survival and apparent cures in mice receiving the DRibble cancer vaccine and anti-OX40.

A second break came when preclinical work from the Earle A. Chiles Research Institute showed that simultaneous combinations of anti-OX40 and anti-PD-1 could be detrimental to treatment, whereas delayed administration of anti-PD-1 could significantly increase apparent cures in a difficult to treat breast cancer model. These data led to the current clinical trial design, with delivery of anti-PD-1 being delayed until the third vaccine.

"While we are excited to be starting this trial with partners at three Cancer Centers in the USA", Fox said, "we hope to expand this trial, and initiate additional studies that employ DPV-001 in combination with traditional cancer treatments, to other parts of the world in the future."

UbiVac considers the path forward is clear. Data from several hundred peer-reviewed publications identifies that patient’s whose immune systems are not turned on to recognize their cancer will respond significantly less well to all forms of cancer treatment. UbiVac believes that DPV-001 provides a treatment which, in combination with other treatments, can engage a patient’s immune system and may improve patient outcome.

Dr. Fox will also discuss a second triplet cancer immunotherapy under development at the Earle A. Chiles Research Institute, a division of the Providence Cancer Institute, in Portland, Oregon, for investigation as treatment for advanced head and neck squamous cell cancer (HNSCC). This trial also plans to use DPV-001 to prime an immune response to a spectrum of HNSCC antigens, a T cell agonist, anti-GITR, and a checkpoint blocker.

Award Number R44CA121612 from the National Cancer Institute, NIH, supported the research and clinical trial of DPV-001 as adjuvant treatment for patients with NSCLC. That trial characterized DPV-001’s safety profile, drug formulation and ability to induce immune responses to a broad spectrum of shared cancer antigens, thus providing the proof of concept for additional trials. This content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

UbiVac is a privately held, clinical stage immunotherapy company engaged in the research and development of therapeutic vaccines to combat cancer. With innovative, first-in-class platform technology that couples an off-the-shelf DC-targeted cancer vaccine with more than 100 cancer antigens for most adenocarcinomas and squamous cell cancers, plus multiple TLR/NOD agonists and DAMPs that are effective at inducing anti-cancer immune responses. UbiVac believes that DPV-001 is highly complementary to current and developing immunotherapy, chemotherapy and small molecule drug portfolios. UbiVac also has a pipeline of vaccines under development to prevent cancer in patients at high risk of developing disease and for those that have failed to respond to anti-PD-1/anti-PD-L1. Founded by Dr. Bernard A. Fox, Dr. Hong-Ming Hu and Mr. Bernard A. Fox, III, in Portland, Oregon in 2005, UbiVac is a spinout of the Robert W. Franz Cancer Center, Earle A. Chiles Research Institute at Providence Portland Medical Center.

On September 24, 2020 Theralase Technologies Inc. ("Theralase" or "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company dedicated to the research and development of light activated Photo Dynamic Compounds ("PDC") and associated drug formulations, reported that McGill University Health Centre ("MUHC") has re-commenced new patient enrollment and treatment in the Company’s Phase II Non-Muscle Invasive Bladder Cancer ("NMIBC") clinical study ("Study II") (Press release, Theralase, SEP 24, 2020, View Source [SID1234569759]).

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Study II has enrolled and treated 12 patients to date. Out of the 7 patients that are eligible to receive the second treatment, 5 have been treated and 2 are pending. 2 out of the last 5 patients treated for the second time have been treated with the optimized Study II treatment, which will also be the case for the 2 patients that are pending their second treatment.

Efficacy to date at the 90 day assessment includes:

3 out of 12 patients (25%) have demonstrated a Complete Response ("CR") (Negative cystoscopy and negative (including atypical) urine cytology
3 out of 12 patients (25%) have demonstrated a Partial Response ("PR") (2 patients with negative cystoscopy and positive urine cytology and 1 patient with positive cystoscopy and negative urine cytology), with 2 of these patients having received second treatment.
The Company is in advanced discussions with the US based Trial Management Organization to potentially launch 5 clinical study sites in the United States in 4Q2020, subject to the US economy successfully recovering from the COVID-19 pandemic. If launched in 4Q2020, the Company would expect Study II patient enrollment and treatment in 1Q2021.

Theralase recently submitted an application to the Food and Drug Administration ("FDA") for Fast Track Approval ("FTA"). The application was denied by the FDA, citing certain improvements required to the application. Theralase has revised the FTA application and has resubmitted it to the FDA. Subject to successful FDA approval of the FTA application, Theralase should be in a position to receive FDA FTA approval in 4Q2020.

Shawn Shirazi, Ph.D., Chief Executive Officer of Theralase stated, "Theralase is pleased that all four Canadian clinical study sites are open for new patient enrollment and we are gearing up to launch 5 additional clinical study sites in the US. We are now able to focus on achieving our interim milestone of treating 20 to 25 patients that could support a Breakthrough Designation application to the FDA. The clinical data collected on the first twelve patients treated shows a favorable clinical response after a single PDT treatment, which the Company expects will improve due to the implemented Study II treatment optimization."

About Study II

Study II utilizes the Therapeutic Dose (0.70 mg/cm2) of TLD-1433 and is focused on the enrollment and treatment of approximately 100 Bacillus Calmete Guérin ("BCG")-Unresponsive NMIBC patients presenting with Carcinoma In-Situ ("CIS") in up to 20 clinical study sites located in Canada and the US.

Study II has a:

Primary endpoint of efficacy (defined by CR at any point in time
Secondary endpoint of duration of CR at 360 days post-initial CR (approximately 450 days post initial Study treatment, assuming CR is achieved at the 90 day assessment)
Tertiary endpoint of safety measured by incidence and severity of Adverse Events ("AEs") grade 4 or higher that do not resolve within 450 days post-initial treatment
The FDA, in its 2018 guidance to industry has stated that, "For single-arm trials of patients with BCG-unresponsive disease, the FDA defines a CR as at least one of the following:

Negative cystoscopy and negative (including atypical) urine cytology
Positive cystoscopy with biopsy-proven benign or low-grade NMIBC and negative cytology
For intravesical therapies without systemic toxicity, the FDA includes, in the definition of a CR, negative cystoscopy with malignant urine cytology, if cancer is found in the upper tract or prostatic urethra and random bladder biopsies are negative.
Intravesical instillation does not deliver the investigational drug to the upper tract or prostatic urethra; therefore, the development of disease in these areas cannot be attributed to a lack of activity of the investigational drug. Thus, sponsors can consider patients with new malignant lesions of the upper tract or prostatic urethra, who have received intravesical therapy to have achieved a CR in the primary analysis; however, sponsors should record these lesions and conduct sensitivity analyses in which these patients are not considered to have achieved a CR."1

On September 24, 2020 Oncoheroes Biosciences, a biotech focused on advancing new therapies for childhood cancer, reported that the United States Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to volasertib, an investigational treatment for rhabdomyosarcoma (Press release, Oncoheroes Biosciences, SEP 24, 2020, View Source [SID1234568287]).

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Key Points
Rare Pediatric Disease Designation qualifies Oncoheroes to receive fast track review, and a priority review voucher (PRV) at the time of marketing approval of volasertib.
PRV holders can benefit from an expedited six-month review of a new drug application for any disease by the FDA.
PRVs are transferable to other sponsor companies and historically have had a selling price range of USD 67 to 350 million.
The US FDA actively supports companies that develop drugs for rare diseases, defined as diseases affecting less than 200,000 Americans. One of the relevant programs created by the agency is for rare diseases primarily affecting individuals younger than 18 years old, called the Rare Pediatric Disease Designation (RPDD) which comes with the related priority review voucher opportunity

"We are delighted that volasertib has been awarded with RPDD by the FDA. This recognition acknowledges the unmet medical need for better treatments for children and adolescents with rhabdomyosarcoma. We hope this big news will allow Oncoheroes to speed up the drug development process of volasertib," stated Ricardo Garcia, Oncoheroes’ Founder and CEO.

Upon drug approval, the RPDD may provide substantial financial incentives by making companies eligible for a Priority Review Voucher (PRV) that is fully transferable. The PRV grants accelerated FDA review of a drug candidate, for any indication, reducing the review period to 6 months and potentially gaining early market access. To date, 12 out of 25 PRVs received for pediatric indications have been sold for a cumulative sale price of USD 1.6 billion.

"Current rhabdomyosarcoma treatments are based on decades-old therapies and generally lack efficacy against the most aggressive subtypes of the disease, for which the 5-year survival rate is currently 20-30%. We are excited about upcoming clinical studies and we hope that volasertib could be a game-changer for rhabdomyosarcoma patients," explained Cesare Spadoni, PhD, Oncoheroes’ Founder and COO.

Around 500 new patients each year in the US are diagnosed with rhabdomyosarcoma, an aggressive and highly malignant form of cancer (soft tissue sarcoma) that develops from skeletal muscle cells that have failed to fully differentiate. There is a clear unmet medical need for the treatment of the most aggressive forms of this disease.

Volasertib is an inhibitor of Polo-like-kinase 1 (PLK1), an enzyme known to be involved in disease progression in a number of cancers. The compound was originally discovered and developed by Boehringer Ingelheim for the treatment of Acute Myeloid Leukemia until the company decided to discontinue the compound for strategic reasons. Meanwhile, independent academic groups generated strong data in support of further development of volasertib for rhabdomyosarcoma and, possibly, a few other pediatric cancer indications. In 2019, Oncoheroes in-licensed volasertib from Boehringer Ingelheim to continue the clinical development of this drug candidate for the benefit of younger cancer patients.

Preclinical research in Rhabdomyosarcoma
A number of publications highlight the potential of volasertib in rhabdomyosarcoma. High PLK1 expression has been associated with poor prognosis in a number of cancers, including rhabdomyosarcoma. It was shown that the drug may have a specific anti-cancer effect in this disease, which is driven by the PAX3-FOXO1 fusion protein in a large subset of patients. PAX3-FOXO1 is a challenging drug target. However, it was shown that PLK1 inhibition by volasertib reduces the stability of this fusion protein leading to its degradation and cancer growth inhibition in PDX models. Interestingly, in vivo data also point to a strong synergy between volasertib and vincristine, a drug already in the standard treatment protocol for rhabdomyosarcoma. Most of these data were generated by European laboratories that are part of the Innovative Therapies for Children with Cancer (ITCC) consortium. Oncoheroes is planning to collaborate with ITCC for the clinical development of volasertib.

On September 24, 2020 Flagship Pioneering reported the launch of Valo Health, LLC (Valo), a drug discovery technology company setting out to transform how oncology, neurodegenerative, and cardiovascular disease drugs are created, developed, and approved (Press release, Flagship Ventures, SEP 24, 2020, View Source [SID1234567634]).

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Founded and initially capitalized by Flagship Pioneering in 2019, Valo received additional funding from investors, bringing total financing to nearly $100m. Valo’s Opal Computational PlatformTM is a first-of-its-kind, end-to-end drug development platform, which integrates machine learning, cloud computing, and data to create an entirely new drug discovery and development process.

"New biological and chemical experimental platforms and the application of advanced computing to integrate human data can shave years off drug development and improve the likelihood of success" said Noubar Afeyan, Ph.D., founder and CEO of Flagship Pioneering. "In a pharmaceutical industry where failure is too often the rule, our goal with Valo is to change how drugs are developed and to bring more predictive confidence to clinical trial design and execution."

Valo’s Opal Computational PlatformTM allows the company’s scientists to analyze human data to uncover previously unsuspected associations between genetic markers and disease with a human-centric framing, enabling the integrated discovery and development of proprietary new molecules.

"At the core of Valo’s approach is a recognition that human-centric data coupled with leading-edge compute can enable a transformation in how drugs are created, reducing cost and time, while increasing confidence," said Valo CEO David Berry, who is also a General Partner at Flagship Pioneering. "We founded Valo with the goal of redefining the drug development process so that we can treat some of the world’s most challenging illnesses more quickly and more effectively than ever before."

During its first year, Valo completed two major acquisitions to help drive the company’s strategy. Numerate, acquired in September 2019, and assets from FORMA Therapeutics, acquired in March 2020. Numerate’s engineers, now part of Valo’s team, created a platform with over 30,000 models and over 70 trillion molecules that powered more than 25 drug programs. Through FORMA Therapeutics, Valo acquired a highly skilled team, research and development compounds and libraries, a global intellectual property portfolio, and two early discovery labs.

"Valo is taking a groundbreaking approach to solving some of the most complex and dynamic issues in drug development," said Ron Hovsepian, Valo Board Chairman, and Flagship Executive Partner. "David Berry’s leadership as an innovator positions Valo to shape how the next generation of drugs will come to market. We are excited to see what comes next."