On March 22, 2021 Nuformix plc (LSE:NFX), a pharmaceutical development company targeting unmet medical needs in fibrosis and oncology via drug repurposing, reported that Oxilio Ltd ("Oxilio") has exercised its option to acquire a licence for NXP001 (a proprietary new form of aprepitant) for oncology indications (Press release, Nuformix, MAR 22, 2021, View Source [SID1234621610]).

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· On 23 September 2020, Nuformix granted an exclusive option to Oxilio, a private pharmaceutical development company, to license NXP001 globally for oncology indications. This triggered the first upfront payment and the option period ran until 23 March 2021
· Nuformix and Oxilio will now work together to finalise a global licensing agreement for NXP001. Once this has been agreed, this will trigger a second undisclosed upfront payment, payable to Nuformix. Under this agreement, Nuformix will license its patent estate and know-how on NXP001 in return for development milestone payments and a royalty on any future net sales, capped at £2m per annum

Dr Anne Brindley, CEO of Nuformix, said: "We are very pleased that Oxilio has exercised its option for NXP001, allowing them to continue the development of NXP001 in oncology. We will now look to finalise the licensing agreement with Oxilio that will provide Nuformix with near term revenue and an opportunity to benefit from the upside of the significant global oncology market. This successful outcome endorses the Nuformix strategy of identifying new improved forms of existing drugs for repurposing into new indications with early-stage licensing during the pre-clinical or Phase 1 stages of development."

Dr Simon Yaxley, Co-Founder and Director of Oxilio said: "Exercising our option gives us the opportunity to continue to develop NXP001 in line with our approach to optimise the delivery of our drug for the effective treatment of certain cancers. We look forward to continuing our collaboration with Nuformix to realise the huge potential of this approach."

About NXP001
NXP001 is a proprietary new form of the drug aprepitant that is currently marketed as a product in the oncology supportive care setting (chemotherapy induced nausea and vomiting). A disadvantage of aprepitant is that its suboptimal properties necessitate a complex formulation. Nuformix discovered new forms of aprepitant (NXP001) with improved properties and it has granted patents on its new forms.

About NXP001 and Oxilio
Oxilio will develop and seek to exploit NXP001 globally for the treatment of cancer. Cancer is one of the world’s primary causes of death. The total worldwide annual economic cost of cancer is substantially in excess of US$1 trillion. Traditional cancer treatments such as surgery, chemotherapy and radiotherapy are expensive and often have debilitating consequences for the patient. Costs of cancer drug treatments are also increasing, in large part due to the expense of taking new 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 alleviating the current dilemma of a shortage of effective drug candidates that have potential as 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 is a less risky development route with substantially lower associated development costs. The agreement with Nuformix allows Oxilio to focus on developing rapidly a unique formulation and dosage form with NXP001 and progressing into the clinic.

Oxilio is a privately held pharmaceutical development company focused on repurposing known drugs for the treatment of cancer through a programme of corporate alliances coupled with rapid proof of concept clinical development.

On March 22, 2021 Aura Biosciences, a clinical-stage oncology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported the closing of an oversubscribed $80 million financing (Press release, Aura Biosciences, MAR 22, 2021, View Source [SID1234576943]). The financing was led by Matrix Capital Management and Surveyor Capital (a Citadel company) with participation from new investors, including Rock Springs Capital, Adage Capital Management LP and Velosity Capital. Existing investors Medicxi, Advent Life Sciences, Lundbeckfonden Ventures, Arix Bioscience, Chiesi Ventures, Ysios Capital and Columbus Venture Partners also participated in the round.

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Aura intends to use the proceeds from this financing to advance the clinical development of its VDC technology platform, including the pivotal Phase 3 program for AU-011, the Company’s lead candidate in development for the first line treatment of choroidal melanoma, and ongoing research for additional programs in ocular oncology, as well as expanding the VDC technology into bladder cancer, the first non-ophthalmic solid tumor indication.

"Aura is pioneering the development of a new class of targeted therapies for life-threatening cancers with our novel VDC technology platform. This funding from a syndicate of distinguished investors enables us to advance AU-011 into a pivotal Phase 3 program for the first line treatment of choroidal melanoma, a rare, life- and vision-threatening form of cancer with no drugs approved. It also allows us to continue to expand the reach of our VDC technology in additional ocular oncology indications and in the treatment of solid tumors like bladder cancer where there is a high unmet medical need for better targeted therapies to treat early and reduce the incidence of metastasis," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.

In connection with this financing, Karan Takhar, Senior Managing Director of Matrix Capital Management, will join Aura’s Board of Directors.

Mr. Takhar said, "Matrix believes in the long-term potential of Aura’s VDC technology to further strengthen the Company’s position as a leader in ocular oncology and beyond within other types of cancers in need of better treatment options. We look forward to supporting Aura’s leadership team through this next stage of pipeline growth and transition into late-stage development with the commencement of the AU-011 pivotal program."

About AU-011 (belzupacap sarotalocan)

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparan sulphate proteoglycans (HSPGs) that are modified and overexpressed on the tumor cell surface of choroidal melanoma cells (and other tumors) and delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant melanoma cells with a pro-immunogenic cell death that is believed to activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. The possibility of early treatment intervention and the activation of the immune system could lead to a reduction in the metastatic rate for patients with this life-threatening disease. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma has been granted Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration and is currently in Phase 2 clinical development.

On March 22, 2021 Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the "Company"), a clinical-stage macrophage reprogramming immunotherapy company targeting diseased macrophages in patients with sepsis, COVID-19 and solid tumors, together with Yale Cancer Center, a National Cancer Institute-designated comprehensive cancer center, reported a research collaboration for the assessment of the potential of Allocetra to enhance the activity of checkpoint inhibitors in solid tumors (Press release, Enlivex Therapeutics, MAR 22, 2021, View Source [SID1234576961]). Allocetra is a macrophage-reprogramming immunotherapy product candidate currently in clinical development by Enlivex, as, among other things, a potential therapy in combination with approved immune checkpoint inhibitors for hard-to-treat solid tumors.

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Under the strategic collaboration, the parties intend to develop and execute pre-clinical programs to investigate the potential synergies between Allocetra and commercially-approved checkpoint inhibitor therapies for select solid cancers.

The Principal Investigator for this collaboration is Dr. Vish Muthusamy, PhD, Director of the Yale Center for Precision Cancer Modeling. Dr. Muthusamy is a cancer biologist with expertise in preclinical investigation of candidate cancer therapies. His center has developed in vivo tumor models to investigate pharmacological evaluation of drugs. Recently, Dr. Muthusamy worked in close collaboration several academic investigators and pharmaceutical companies to develop cancer therapeutics that are in various stages of clinical development. The collaboration’s scientific advisor is Dr. Marcus W. Bosenberg, MD, PhD, Co-Leader of the Genomics, Genetics and Epigenetics Program at Yale Cancer Center. On behalf of the Yale team, Dr. Bosenberg said "At Yale we have been interested in trying to understand the process of macrophage reprogramming for some time, and we are excited about studying the effects of Allocetra in potentially recruiting anti-tumor macrophages in the tumor environment and characterize the effects."

Dror Mevorach, M.D., Chief Medical Officer of Enlivex, commented, "We are excited to collaborate with the distinguished research and clinical teams at Yale. Allocetra may have a rebalancing effect on the typically immunosuppressive tumor microenvironment, potentially by facilitating the conversion of pro-tumor macrophage populations to anti-tumor populations. Together, we plan to investigate the potential of AllocetraTM to synergistically combine with commercially available checkpoint inhibitors for the treatment of solid tumors."

Oren Hershkovitz, Ph.D., CEO of Enlivex, stated, "We believe the researchers and clinicians at Yale are world-class and ideal partners as we work to realize Allocetra’s potential. We are pleased to formulate a strategic collaboration with Yale for the development of Allocetra as a potentially key component of combination therapies for solid tumors."

Allocetra is currently in clinical development for acute life-threatening immune-mediated diseases, such as sepsis and COVID-19. Enlivex recently reported positive top-line results in 21 patients from Phase Ib and Phase II investigator-initiated trials in COVID-19 patients in severe/critical condition. The Company has also previously reported positive results from a Phase Ib investigator-initiated trial in 10 sepsis patients and plans to initiate a controlled, randomized, Phase IIb study in sepsis during the first quarter of 2021.

On March 22, 2021 Sirtex Medical ("Sirtex"), a leading manufacturer of targeted liver cancer therapies, reported full FDA approval of the DOORwaY90 Study, a trial evaluating the safety and efficacy of selective internal radiation therapy (SIRT) using SIR-Spheres Y-90 resin microspheres in patients with unresectable hepatocellular carcinoma (HCC) (Press release, Sirtex Medical, MAR 22, 2021, View Source [SID1234576980]).

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Unique to other recently published Y-90 studies, DOORwaY90, which stands for "Duration of Objective Response with Arterial Y-90," is the first prospective, multicenter study to utilize and delineate personalized dosimetry treatment planning and to define actionable post-treatment dosimetric verification for endpoint assessment. The study will assess the duration of response (DoR) and objective response rate (ORR) of SIR-Spheres.

Outside the United States, SIR-Spheres are indicated for the treatment of patients with advanced non-operable liver cancer, including HCC. "Our therapy is used for treatment in HCC in more than 50 countries, with years of safety and efficacy," said Kevin Smith, Chief Executive Officer at Sirtex. "The DOORwaY90 Study has the potential to expand the FDA-approved indication for use of SIR-Spheres in the U.S., which would mark an incredible achievement in patient care."

The DOORwaY90 Study is being led by co-principal investigators Cheenu Kappadath, PhD, and Dr. Armeen Mahvash. "We are honored to participate in this important study that could greatly impact the treatment of HCC patients in the United States," noted Dr. Mahvash, Professor in the Department of Interventional Radiology Division of Diagnostic Imaging at the University of Texas MD Anderson Cancer Center. "We look forward to working closely with Sirtex in executing and reporting the findings of DOORwaY90."

DOORwaY90 is a 15-center, 100-patient, U.S.-based open label, single arm study run in accordance with Good Clinical Practice (cGCP). The study population consists of patients with Barcelona Clinic Liver Cancer (BCLC) Stage A, B1 and B2 who are not eligible for resection or ablation at the time of study entry. For each patient, an eligibility review committee will review diagnostic imaging and confirm final eligibility and treatment planning prior to treatment. Enrollment is expected to begin in early Q2 2021.

HCC is often diagnosed when potentially curative resection or transplantation is not feasible. SIRT has the potential to deliver a lethal dose of radiation to hepatic tumors, while sparing surrounding healthy liver tissue. In countries outside the U.S., SIRT has been successfully used to bridge patients to transplantation or downstage HCC to within transplantation criteria or resection.

On March 22, 2021 Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) reported that the Company has entered into a multi-RNA target alliance with Takeda Pharmaceutical Company Limited ("Takeda") with the goal to discover and develop RNA targeting small molecule therapeutics for highly attractive targets that are difficult to address via more conventional approaches (Press release, Evotec, MAR 22, 2021, View Source;announcements/press-releases/p/evotec-and-takeda-enter-strategic-rna-targeting-drug-discovery-and-development-alliance-6038 [SID1234576944]).

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Evotec and Takeda will jointly identify and develop small molecules targeting a range of RNA targets aligned with Takeda’s research and development areas. The collaboration will leverage Evotec’s extensive RNA targeting platform to optimally identify promising RNA sequences to target with small molecule ligands that can be developed into potentially first-in-class therapeutics.

Under the terms of the agreement, Evotec will receive significant research funding and will be eligible to receive discovery, pre-clinical, clinical, commercial and sales milestone payments of up to US$ 160 m per programme. Additionally, Evotec is entitled to tiered royalties on net sales of any products resulting from the collaboration.

Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "Many highly validated targets have proven to be intractable via conventional protein targeting approaches. For this reason, Evotec has been pioneering RNA targeting strategies and approaches for quite some time. We are very excited about the opportunity to collaborate with Takeda in this field as both companies share the vision to jointly develop small molecule therapeutics against high value RNA targets that will deliver long awaited therapeutics."

"Takeda recognizes targeting RNA with small molecules as a promising new modality that has tremendous potential for much needed medicines for patients through modulating historically undruggable targets", said Dr Larry Hamann, Head, Drug Discovery Sciences, Takeda. "We are excited to be working with Evotec and their impressive capabilities."