On April 21, 2020 Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for 67Cu-SARTATE, a therapy for the clinical management of neuroblastoma (Press release, Clarity Pharmaceuticals, APR 21, 2020, View Source [SID1234556447]).

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Neuroblastoma most often occurs in children younger than 5 years of age and presents when the tumour grows and causes symptoms. It is the most common type of cancer to be diagnosed in the first year of life and accounts for around 15% of paediatric cancer mortality. High-risk neuroblastoma accounts for approximately 45% of all neuroblastoma cases. Patients with high-risk neuroblastoma have the lowest 5-year survival rates at 40%-50%.1

FDA grants ODDs to facilitate the development of investigational therapies intended to treat, diagnose or prevent rare diseases affecting fewer than 200,000 people in the United States. The designation allows Clarity Pharmaceuticals to qualify for a number of incentives, including: seven years of market exclusivity upon regulatory approval, if received; potential tax credits on US clinical costs; exemptions from certain administrative FDA fees; and eligibility for grants to fund future clinical work.

Dr Alan Taylor, Clarity’s Executive Chairman, commented, "At Clarity, we are especially passionate about the development of SARTATE in neuroblastoma. At present, prognosis of high-risk neuroblastoma patients remains unfavourable, despite intensive, multimodal therapy. Also, the effectiveness of current neuroblastoma treatment strategies, such as chemotherapy, radiation, and surgery, is limited in late-stage disease due to patients’ numerous metastases. It is clear that improvements in current treatment approaches are needed to overcome poor outcomes for the patients."

"ODD status will potentially provide a number of advantages for the development of SARTATE for the treatment of neuroblastoma and allow us to progress more swiftly to our ultimate goal of better treatment of children and adults with cancer."

On April 20, 2020 Phost’in Therapeutics (Montpellier, France), a biopharmaceutical company focused on the discovery and development of N-glycosylation inhibitors, reported that it has successfully secured a €10.3 million Series A round, from an international syndicate of LifeScience and Innovation Investors led by Remiges Ventures and completed by ANRI and IrdiSoridec (Press release, Phost’in, APR 20, 2020, View Source [SID1234560965]).

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On April 20, 2020 Zikani Therapeutics, a company dedicated to leveraging its unique TURBO-ZM platform to develop novel ribosome modulating agents (RMAs) for the treatment of rare, nonsense mutation-driven diseases, reported the close of a $7.5 million Series A-1 financing (Press release, Zikani Therapeutics, APR 20, 2020, View Source [SID1234558245]). Advent Life Sciences, Gurnet Point Capital and Roche Venture Fund supported the funding.

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"We’re pleased to support Zikani as an emerging company that is using the strength of its science and a refined strategy to develop novel ribosome modulators for rare diseases caused by nonsense mutations," said Raj Parekh of Advent Life Sciences.

Zikani will use the proceeds to advance its current nonsense mutation readthrough programs from lead optimization to candidate selection. The company has demonstrated positive, pre-clinical data in multiple disease areas, including: APC mutant colon cancer, familial adenomatous polyposis (FAP), class 1 cystic fibrosis, and recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB).

"Zikani has generated promising data that validates its TURBO-ZM platform and addresses a completely unmet need in oncology therapeutics: restoring expression of a major tumor suppressor gene," said Keith Flaherty, M.D., Director of Clinical Research at Massachusetts General Hospital Cancer Center and a senior advisor to Zikani.

Ribosomal RNAs form the translation machinery that generates function proteins from genetic sequencies. Ribosome modulation provides a therapeutic approach to addressing a number of diseases, but the development of disease-specific ribosome modulators has been a challenge. Zikani’s proprietary platform TURBO-ZM allows rapid synthesis of novel RMAs that can be optimized to target the human ribosome in a disease specific manner.

"We’re highly encouraged by the data we’ve generated to date and are working rapidly to advance compounds into pre-clinical development. This financing, particularly in this volatile financial market, is a vote of confidence for our promising technology platform. As we look ahead, our focus is to maximize the funding and take the steps necessary to generate greater recognition for RMAs and form collaborations with partners that will benefit from our scientific platform," said Sumit Aggarwal, President and CEO, Zikani Therapeutics.

On April 20, 2020 Epsilogen (formerly IGEM Therapeutics), a global leader in the development of immunoglobulin E (IgE) antibodies to treat cancer, reported that it has in-licenced exclusive global rights to MOv18 IgE, an anti-folate receptor alpha IgE antibody from King’s College London (KCL) (Press release, IGEM Therapeutics, APR 20, 2020, View Source [SID1234556608]). Financial terms of the agreement have not been disclosed.

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The antibody, which was developed at KCL in collaboration with Cancer Research UK, is the first therapeutic IgE antibody to enter clinical trials. Cancer Research UK is funding, sponsoring and conducting an on-going phase I clinical trial in patients with advanced cancer expressing folate receptor alpha (FR alpha). As separately announced today, interim data from this trial will be presented at the forthcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting I which begins on 27 April 2020. Folate receptor alpha (FR alpha) is selectively expressed most commonly on the surface of ovarian cancer cells, making it a promising target for IgE antibody therapy for this cancer which has relatively few effective treatment alternatives.

Epsilogen will continue the further clinical development of MOv18 IgE and has put in place a comprehensive manufacturing programme with a leading contract development and manufacturing organisation. Funding for this manufacturing work has been provided by a further £3 million Series A2 investment from Epsilogen’s existing investors Epidarex Capital, ALSA Holdings and the UCL Technology Fund.

Dr Tim Wilson, Chief Executive Officer of Epsilogen, said:

"We are very pleased to have in-licensed this promising new therapy which has the potential to target a very serious cancer with relatively few effective treatment alternatives. We are grateful to our scientific founders Professors Sophia Karagiannis and James Spicer of KCL for conducting the early scientific research on MOv18 IgE and also to Cancer Research UK for investing in and developing the product through its early clinical phase. We also wish to thank our investor group for continuing to support us with further financing via the Series A2."

On April 20, 2020 Genome & Co. (KONEX: 314130), a Korean biotechnology company developing innovative therapeutics in immune-oncology, reported on April 20 that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for GEN-001 for combination treatment with avelumab (BAVENCIO) in patients with solid cancers (Press release, Genome & Company, APR 20, 2020, View Source [SID1234556600]).

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Avelumab is an anti-PD-L1 antibody co-developed and co-commercialized by Merck KGaA, Darmstadt, Germany and Pfizer Inc.

With this clearance, Genome & Co. will be the first Asian company to initiate a first-in-human trial of anti-cancer microbiome and anti-PD1/anti-PD-L1 combination treatment as a sole sponsor. The phase 1/1b clinical trial will be initiated at the U.S. clinical sites and the first patient is expected to be enrolled within this year.

"IND clearance from FDA for our first anti-cancer microbiome therapeutic GEN-001 is a very significant milestone as it will transition Genome & Co. into a clinical-stage biotechnology company. We hope to add meaningful value and advancement in the microbiome and immuno-oncology industry with our combinational approach to cancer patients who have progressed on prior anti-PD1/anti-PD-L1 therapy," said Park Han-soo, chief technical officer of Genome & Co.

Genome & Co CEO Pae Ji-soo said, "This IND approval is a meaningful corporate milestone and a critical step forward to achieving new arrangements in strategic partnering. We are indeed looking forward to further investigating how our clinical data will be translated into our cancer patients. I thank all the members and partners of Genome & Co. for dedicating themselves to accomplishing this milestone."

In January this year, Genome & Co. had entered into a clinical trial collaboration and supply agreement with Merck KGaA, Darmstadt, Germany and Pfizer Inc. to evaluate the safety, tolerability, biological and clinical activities of GEN-001 therapy in combination with avelumab in multiple cancer indications. The combination trial is designed to be a first-in-human study including dose escalation and expansion cohorts to evaluate the safety and preliminary efficacy.

GEN-001 is an oral microbiome therapeutic candidate developed to have immune modulating activities, resulting in potential partnership with immune checkpoint inhibitors. GEN-001 consists of a single strain bacteria isolated from gut of healthy human volunteers that has been shown to activate dendritic cells, macrophages and T cell response. In preclinical studies, GEN-001 has shown optimal safety margin and synergistic effects in combination with immune checkpoint inhibitors by enhancing the effect of suppressing the growth of both immune checkpoint inhibitor sensitive and resistant tumor models.

Genome & Co. is a clinical stage biotechnology company based in South Korea that focuses on discovering and developing the next waves of innovative therapeutics in immuno-oncology through diverse modalities of microbiome, novel target immune checkpoint inhibitors and fusion proteins to fulfill the unmet needs of cancer patients.