On October 12, 2020 Kanaph Therapeutics, a leading biotech in oncology, auto-immune, and eye diseases with large molecules and small molecules, reported that recently completed their Series B funding of 21M USD in South Korea (Press release, Kanaph Therapeutics, OCT 12, 2020, View Source [SID1234568361]). In this round, a large Korean pharma company, GC Pharma, and venture capital funds such as Kolon Investment, Timefolio Asset Management and more participated in the fund raising. In addition to the 2M USD angel funding and 6M USD Series A funding, Kanaph has raised a total of 30M USD to date.

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Kanaph’s front runners are bi-specifics from their TMEkineTM platform for immuno-oncology, and a bi-specific Fc-fusion inhibiting both alternative complement and angiogenesis pathways for the treatment of retinal disorders. Their small molecule pipelines include the development of novel small molecules targeting specific mutational signaling pathways or removing immune-suppressive environment created by tumors. Kanaph focuses on three small molecule programs in this regard: a 4th generation EGFR inhibitor targeting C797S, a dual EP2/4 inhibitor and an undisclosed target in the KRAS pathway, where the first two are entering the preclinical stage in early 2021.

‘TMEkineTM’, an antibody platform, is built on technologies licensed in from top US universities and is integrated into an antibody-cytokine bispecific platform that can overcome the largest unmet needs of existing immuno-oncology therapies or treat non-responsive patient groups. TMEkineTM molecules are in line with the recent trend in the field of immuno-oncology, which has been the conversion of tumors that do not respond to immunotherapies, also known as cold tumors, into hot tumors. The platform can also be expanded into a wide range different indications.

The management team at Kanaph have extensive research and drug development experience in companies such as Genentech and Amgen. The CEO of Kanaph, Byoung Chul Lee has a strong track record in antibody drug discovery and development stemming from his experience in Genentech, 23andMe and Santen.

This round of funding will go towards expediting the clinical development of Kanaph’s pipelines. Candidates eligible are TMEkineTM molecules for immuno-oncology and bi-specific Fc fusions for the treatment of retinal diseases. A CDO agreement was recently signed with Samsung Biologics for the development of KNP-301 (retinal disease therapy) for the clinic.

The funds will provide a basis for completing preclinical studies for a number of pipelines at the end of this year or the beginning of next year and they will be licensed out once their preclinical packages are ready for the next steps.

On October 12, 2020 Glycostem Therapeutics, a leading clinical-stage company focused on the development of therapeutic off-the-shelf Natural Killer (NK) cells, reported it has received the FDA’s Orphan Drug Designation (ODD) for treatment of Multiple Myeloma (MM) patients with its investigational product oNKord (Press release, Glycostem Therapeutics, OCT 12, 2020, View Source [SID1234568359]). The designation will provide Glycostem with certain incentives, like eligibility for 7 years of market exclusivity and clear FDA guidance on specific aspects of development for rare diseases. These pave an accelerated path towards market access and treatment of patients suffering from this relatively rare form of cancer.

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oNKord is Glycostem’s first-generation off-the-shelf Natural Killer (NK) cellular immunotherapy product. Over the coming months, AML patients will receive this form of treatment as part of a phase I-IIa (pivotal) trial in AML. A phase II trial for MM patients is expected to start in 2021. This makes Glycostem one of the frontrunners in this promising field of cellular immunotherapy.

"Since 2012 we have been pioneers in the field of developing and manufacturing off-the-shelf Natural Killer cell therapy products for cancer treatment. In 2020 we’re entering a new and exciting phase," says Troels Jordansen, CEO of Glycostem. "It is great to experience that after receiving FDA and EMA ODD designation for AML, the FDA has also granted us this designation for MM. This allows us to accelerate oNKord’s access to the US market and our ultimate ambition: curing cancer."

Multiple Myeloma (MM)

MM is the second most common blood cancer, accounting for 15% of blood cancers, and 2% of all cancers. In the US alone it affects more than 130,000 patients; approximately 32,000 Americans are diagnosed with MM each year. MM occurs in infection-fighting plasma cells (a type of white blood cell) found in the bone marrow. These cancerous cells multiply, produce an abnormal protein and push out other healthy blood cells from the bone marrow.

Orphan Drug Designation

The FDA grants orphan drug designation to drugs and biologics for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the US. The designation allows manufacturers to qualify for various incentives, including exemption of FDA application fees), tax credits for qualified clinical trials and be eligible for 7 years of market exclusivity after regulatory approval.

On October 12, 2020 BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, reported Stacy Lindborg, Ph.D., Executive Vice President and Head of Global Clinical Research, will deliver a presentation at the 2020 Cell & Gene Meeting on the Mesa, being held virtually October 12-16, 2020 (Press release, BrainStorm Cell Therapeutics, OCT 12, 2020, View Source;gene-meeting-on-the-mesa-301150038.html [SID1234568358]).

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Dr. Lindborg’s presentation will be in the form of an on-demand webinar that will be available beginning today. Those who wish to listen to the presentation are required to register here. At the conclusion of the 2020 Cell & Gene Meeting on the Mesa, a copy of the presentation will also be available in the "Investors and Media" section of the BrainStorm website under Events and Presentations.

About the 2020 Cell & Gene Meeting on the Mesa

The conference will feature 80+ on-demand company presentations by leading public and private companies, highlighting their technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, and tissue engineering. Registrants will have access to 15+ expert-led panels and workshops including a mix of both live and on-demand sessions. The conference will be delivered in a virtual format over the course of five days – October 12-16. There is also a premier partnering system, partneringONE, allowing registrants to plan 1×1 meetings with other attendees. For a list of presenting companies, refer to View Source

On October 12, 2020 KAHR, a cancer immunotherapy company developing novel multifunctional immuno-recruitment proteins, reported that an abstract reporting preclinical data for DSP107, a second generation CD47x41BB targeting compound for the treatment of solid tumors and hematological malignancies, has been accepted for an oral presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020 (Press release, KAHR Medical, OCT 12, 2020, View Source [SID1234568356]).

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"The ASH (Free ASH Whitepaper) Annual Meeting will be an important opportunity to present mechanistic studies and extensive in vitro and in vivo results from our novel CD47x41BB clinical stage drug candidate," said Yaron Pereg, Ph.D., Chief Executive Officer of KAHR. "We look forward to advancing the clinical development of this novel therapy."

The following abstract will be posted on the ASH (Free ASH Whitepaper) website on November 5, 2020, at 9:00 a.m. ET:

Title: DSP107, a Novel Bi-Functional Fusion Protein That Combines Inhibition of CD47 with Targeted Activation of 4-1BB to Trigger Innate and Adaptive Anticancer Immune Responses

Publication Number: 173
Session Name: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Session Date: Saturday, December 5, 2020
Session Time: 12:00 PM – 1:30 PM
Presentation Time: 12:30 PM

Session Name: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Novel Approaches to Overcome Resistance

About DSP107

DSP107 targets CD47-overexpressing tumors, simultaneously blocking macrophage inhibitory signals and delivering an immune costimulatory signal to tumor antigen-specific, activated T-cells. CD47 is overexpressed on many cancer cells and binds SIRPα on immune phagocytic cells to produce a "don’t eat me" signal. DSP107 binds CD47 on cancer cells, blocking interaction with SIRPα and thus, blocking the "don’t eat me signal". Simultaneously, DSP107 binds 41BB on T-cells, stimulating their activation. These activities lead to targeted immune activation through both macrophage and T-cell mediated tumor destruction.

About DPS107 Phase I/II

A Phase I/II clinical trial to evaluate the safety, pharmacokinetics (PK) and pharmacodynamics (PD) of DSP107 as a monotherapy and in combination with Roche’s PD-L1-blocking checkpoint inhibitor (CPI) atezolizumab (Tecentriq) in patients with advanced solid tumors is currently being activated. The preliminary efficacy of both DSP107 monotherapy and combination therapy with atezolizumab will also be evaluated in patients with advanced non-small-cell lung carcinoma (NSCLC) who progressed after treatment with PD-1/PD-L1 inhibitors. The study will be conducted at multiple centers in the United States under a clinical collaboration with Roche.

On October 12, 2020 Oxular Limited ("Oxular"), a leading retinal therapeutics development company, reported it has received both Rare Paediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (U.S. FDA) for OXU-003, the Company’s proprietary drug in development for the treatment of retinoblastoma (Press release, Oxular, OCT 12, 2020, View Source [SID1234568355]).

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Retinoblastoma is a rare form of eye cancer that usually develops in early childhood, typically before the age of five. This form of cancer develops in the retina, which is the specialised light-sensitive tissue at the back of the eye that detects light and colour. The most common first sign of retinoblastoma is a visible whiteness in the pupil called "cat’s eye reflex" or leukocoria. Other signs and symptoms of retinoblastoma include crossed eyes or eyes that do not point in the same direction (strabismus), a change in eye colour, redness, soreness, or swelling of the eyelids, and blindness or poor vision in the affected eye or eyes.

Retinoblastoma is often curable when it is diagnosed early. However, if it is not treated promptly, this cancer can spread beyond the eye to other parts of the body. This advanced form of retinoblastoma can be life-threatening.

Current treatment options for retinoblastoma include systemic chemotherapy and intra-arterial chemotherapy, which are very invasive procedures and require specialised facilities and hospital stay. Intra-arterial chemotherapy is only available in select centres. These treatments can lead to significant side effects for patients, including neurocognitive impairment, loss of vision and hearing, and life-threatening infections (sepsis) from low blood counts (neutropenia).

Oxular’s OXU-003 programme for the treatment of retinoblastoma consists of a proprietary anti-tumour drug which utilises Oxular’s formulation and ocular administration technology to safely deliver a precise amount of drug adjacent to the primary ocular tumour (local or targeted chemotherapy). OXU-003 has been shown to be effective as a stand-alone therapy in preclinical models and is complementary with other agents currently used to treat retinoblastoma. Oxular’s minimally invasive local therapeutic approach is intended to be less risky compared to current treatments, is expected to spare patients from related side effects while preserving vision and can be administered by an ophthalmic surgeons in standard operating theatres without the need of specialised equipment.

Mr. Manoj Parulekar, Paediatric Consultant Ophthalmologist, Birmingham Children’s Hospital, commented:

"I am very pleased to see the U.S. FDA’s acknowledgement of the critical and urgent need to develop an effective, safer and easily accessible treatment for Retinoblastoma which is such a devastating disease. Given the potential severe side effects associated with current treatments, which can have life-long impact on children’s lives, it is important that these patients have another treatment available to them."

Thomas Cavanagh, Chief Executive Officer of Oxular commented:

"We are delighted to receive these Rare Paediatric Disease and Orphan Drug designations, which provide important momentum for our OXU-003 development program, as we expect to enter human clinical trials and generate data within the next two years. OXU-003 is a potential breakthrough therapy that utilises Oxular’s core technology to maximise the opportunity for successful treatment while preserving quality of life for these young patients."

The U.S. FDA grants Rare Paediatric and Orphan Drug designations to drugs intended for the treatment of rare diseases that primarily affect children ages 18 years or younger and fewer than 200,000 persons in the U.S. If a future New Drug Application (NDA) for OXU-003 is approved, Oxular is eligible to receive a Priority Review Voucher that may be sold or transferred.

The first NDA applicant to receive FDA approval for a particular active moiety to treat a particular disease with FDA Orphan Drug designation is entitled to various incentives of the Orphan Drug Act (ODA), including tax credits for qualified clinical testing, waiver of NDA / biologics license application (BLA) user fees, and eligibility for a seven-year exclusive marketing period for that drug and use upon marketing approval.