SQZ Biotech Lines Up an IPO on the NYSE to Fund Cell Therapy R&D

On October 12, 2020 SQZ Biotechnologies, reported that which already has an alliance with Roche focused on developing new cancer cell therapies, is now looking to sell shares to the public to finance its research of potential new treatments for both cancer and infectious disease (Press release, SQZ Biotech, OCT 12, 2020, View Source [SID1234568458]).

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In paperwork filed with securities regulators late last week, SQZ set a preliminary $75 million goal for its IPO. The Watertown, MA-based biotech has applied for a listing on the New York Stock Exchange under the stock symbol "SQZ."

SQZ (pronounced "squeeze") is developing technology intended to improve cell therapy. Current cell therapies get the therapeutic cargo into a cell via an engineered virus or electroporation, a technique that involves zapping a cell’s membranes open with electricity. The latter approach can damage the cell, impeding its function. SQZ also says in its filing that both techniques are used primarily for delivering nucleic acids to certain cell types and they don’t work well for proteins or small molecules.

On the patient side, cell therapy requires "pre-conditioning," a depletion of immune cells to improve the efficacy of the infused cell therapy. Cell therapies also come with warnings of potentially life-threatening safety risks. Furthermore, both electroporation and viral engineering can lead to downstream biological effects.

SQZ says it can avoid limitations of current cell therapies with a proprietary technology that physically squeezes cells through a microfluidic chip, opening the cell membrane and allowing therapeutic cargo to diffuse inside. The biotech says its approach offers a manufacturing advantage—less than 24 hours production time compared to a month or more for current cell therapies. Cell therapies made with SQZ’s technology don’t require patient pre-conditioning, which shortens hospital stays, and the company says its technology can potentially apply to many more diseases.

The SQZ technology was invented by company founder and CEO Armon Sharei based on the research of his team in the laboratories of Klavs Jensen and Robert Langer at MIT. The company has developed three platforms: one targeting antigen-presenting cells (APCs), another for activating antigen carriers (AACs), and a third for tolerating antigen carriers (TACs). SQZ’s initial focus areas are oncology, infectious disease, and immune disorders. SQZ was an Xconomy Awards 2020 finalist in the "Big Idea" category. Sharei explains his company’s technology in this video (SQZ’s portion starts at 18:18).

Roche began its partnership with SQZ in 2015, the same year that the company licensed its technology from MIT, according to the IPO filing. That alliance aimed to use the SQZ technology to develop cancer cell therapies based on B cells, a type of immune cell. In 2018, the partners amended the pact to include APCs. Under the collaboration agreement, the partners jointly select antigen targets. SQZ is responsible for preclinical research and Phase 1 development of cell therapy candidates. After that, Roche has the option to exercise an exclusive global license to APC products from the collaboration on a product-by-product basis. Those rights only cover cancer. For other indications, or for cancer applications that Roche opts not to pursue, SQZ retains all rights.

The most advanced drug candidate in the alliance, SQZ-PBMC-HPV, comes from SQZ’s APC platform. In preclinical research, SQZ says it was able to squeeze antigens targeting the KRAS cancer protein into two sets of human donor cells. In both, the company observed antigen-specific activation of immune cells, according to the IPO filing. Based on the results, SQZ says it believes cell therapies from its APC platform has potential applications in HPV positive tumors and cancers caused by KRAS mutations, among other oncology applications.

The SQZ drug is currently in Phase 1 testing in in patients with locally advanced and metastatic HPV positive tumors. SQZ expects initial monotherapy data from the study will be reported in the first half of next year. Initial Phase 1 data from tests of that drug as part of a combination therapy are expected in the second half of 2021.

Since its formation, SQZ says it has raised more than $166.6 million from equity investments, most recently a $65 million Series D round in May. The company also says that it has received $94 million in upfront and milestone payments from Roche. Polaris Partners is SQZ’s largest shareholder with just over 14 percent of the company, according to the IPO filing. The second largest shareholder with 9.6 percent is Elbrus Investments, followed by AIG DECO Fund with 7.7 percent.

SQZ says it will apply the IPO proceeds toward the completion of Phase 1 development of its lead therapeutic candidate. Some of the IPO cash will support continued development of SQZ-AAC-HPV, compound from its AAC platform that is another potential treatment for HPV positive tumors. The company says it will also use some of the IPO proceeds for the preclinical research needed to support an application to begin human testing of its treatment candidates for infectious diseases.

Rhizen Pharmaceuticals S.A. and Curon Biopharmaceutical Limited Announce an Exclusive Licensing Agreement to Develop and Commercialize Tenalisib, a Dual PI3K Delta and Gamma Inhibitor for Oncology in Greater China

On October 12, 2020 Rhizen Pharmaceuticals S. A. (Rhizen), a Switzerland-based privately held clinical-stage biopharmaceutical company, and Curon Biopharmaceutical Limited (Curon), a clinical-stage innovative biopharmaceutical company with facility in Shanghai reported that they have entered into an exclusive licensing agreement for the development and commercialization of Tenalisib, a dual PI3K delta & gamma inhibitor in the Greater China region (Press release, Rhizen Pharmaceuticals, OCT 12, 2020, View Source [SID1234568376]).

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Under the terms of the agreement, Rhizen will receive an undisclosed upfront cash payment and is eligible to receive additional development and commercial milestone payments with an overall deal value of USD 149.5 million plus double-digit royalties on annual net sales of Tenalisib.

Curon obtains the exclusive development and commercialization rights of Tenalisib for Greater China across all oncology indications, and will lead the clinical development in that territory by leveraging its unique expertise in translational research, clinical development and regulatory registration and its extensive research collaboration experience, to accelerate the development of and regulatory approval of this product in Greater China.

Swaroop Vakkalanka, Ph.D., President and Chief Executive Officer of Rhizen Pharmaceuticals stated, "Emerging human clinical data demonstrates that Tenalisib is a differentiated, next-generation, orally active, dual PI3K delta and gamma inhibitor with an excellent safety profile and promising single-agent activity in haeme malignancies. We believe, Tenalisib’s outstanding safety could allow rational combinations with other approved/investigational agents and enable us to unlock the true potential of this class of drugs. Our partnering with Curon is a first step towards achieving this objective and we look forward to the day this novel drug reaches cancer patients in need of new and safe therapies."

"Tenalisib has demonstrated great efficacy in lymphoma patients with outstanding safety profile, in-licensing this product to China would bring more effective and additional treatment options to Chinese cancer patients and greatly benefit these patients. Meanwhile, this will further enrich our diversified pipeline. We are very happy and look forward to closely collaborating with Rhizen to efficiently develop this molecule into an effective medicine to benefit patients not only in China but also around the world as soon as possible." said Zhihong Chen, Ph.D., President of Curon.

Kun Tao from Yafo Capital acted as financial advisor on this transaction for Rhizen.

About Tenalisib (RP6530):

Tenalisib (RP6530) is a highly selective, next-generation, orally active, dual PI3K delta and gamma inhibitor, that is currently in Phase 2 clinical development for hematological malignancies. Tenalisib has been granted US FDA Fast Track & Orphan-Drug Designations for treatment of relapsed/refractory peripheral T-cell lymphoma and cutaneous T-cell lymphoma (R/R PTCL and R/R CTCL).

Aurum Biosciences Receives £670k in New Equity Funding Round

On October 12, 2020 Aurum Biosciences Ltd (Aurum) reported that it has raised approximately £670k as part of a new round of equity funding (Press release, Aurum Biosciences, OCT 12, 2020, View Source [SID1234568303]). This funding round was supported by Infinion Biopharma Limited (Infinion), the Scottish Investment Bank, Scottish Health Innovations Ltd (SHIL), TRICAPITAL business angel syndicate and individual investors.

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Aurum is a Scottish based clinical stage biopharmaceutical company developing novel oxygen carriers and software for use with MRI. Aurum works closely with the University of Glasgow and NHS Greater Glasgow and Clyde to develop products which it hopes will bring significant lifesaving benefits in the areas of stroke, cardiology, oncology and neuro-degenerative diseases.

These funds which will further extend the work already funded by the Wellcome Trust will be dedicated to the company’s core strategy of developing novel therapies and diagnostics in stroke, using its lead product ABL-101. Aurum aim to deliver a phase 2 clinical trial in stroke patients in Glasgow’s Queen Elizabeth University Hospital.

Jim McGuire, Aurum’s CEO said, "We are delighted to complete this second round with our initial investment partners and bringing in a new investment partner, Infinion. This funding will allow us to extend our work beyond the successfully completed pre-clinical trials where Aurum’s lead product has demonstrated great potential in both stroke treatment and diagnosis. We are especially pleased that Infinion have joined the investment and look forward to them helping to drive Aurum forward with their global expertise in the biopharma sector. There has been few new therapies or diagnostics approved for acute ischaemic stroke in recent years. Our products provide hope for the many patients, carers and healthcare professionals who live with the consequences of this devastating condition."

Commenting on the investment Graham Watson, Executive Chair of SHIL said: "SHIL has been working with Aurum Biosciences for over five years following the successful spin-out from NHS Greater Glasgow and Clyde and University of Glasgow. Significant progress has been made in advancing the diagnosis and treatment of stroke with promising results evident in pre-clinical trials. This is vital work; in Scotland alone stroke remains the third biggest killer and the leading cause of disability. This new investment from SHIL and other investors, combined with ambitious development plans, will support the team to extend their work in stroke, and other conditions; with the ultimate aim of saving and improving lives."

Commenting on the investment Govind Chavada, Managing Director of Infinion said: "Worldwide, cerebrovascular accidents (stroke) are the second leading cause of death and the third leading cause of disability. Infinion remains committed to bring novel therapies to the market and to that end the symbiosis of Infinion and Aurum is the step in the right direction. Aurum’s lead candidate ABL 101 has great potential to change the therapeutic landscape of ischaemic stroke and other disorders where hypoxic tissue damage is the primary driver of disease pathology"

Commenting on the investment Jamie Andrew, Chair of Tricapital said: "Stroke remains the third biggest killer in Scotland and the leading cause of disability and is the second leading cause of death worldwide. TRICAPITAL are delighted to provide this further support that will enable Aurum to continue to develop important new treatments and diagnostics in this vital area of healthcare."

Kanaph Therapeutics Closes 21M USD Series B Funding

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.

FDA grants Glycostem’s oNKord® Orphan Drug Designation for Multiple Myeloma

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.