On May 6, 2021 Sanofi reported that it has entered into a three-year research collaboration with Stanford University School of Medicine. Together, the two organizations and their scientists will work to advance the understanding of immunology and inflammation through open scientific exchange (Press release, Sanofi, MAY 6, 2021, View Source [SID1234579260]). Additionally, Sanofi will provide funding and scientific inputs into projects of mutual interest, crossing multiple therapeutic areas including autoimmune diseases and inflammatory conditions.

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"We look forward to working with some of the most innovative scientists in the human immunology community. Together we will explore groundbreaking concepts and obtain deeper insights into underlying inflammatory disease mechanisms," said Frank Nestle, Global Head of Research and Chief Scientific Officer, Sanofi. "Sanofi’s collaboration with Stanford University aims to transform how autoimmune disorders and inflammatory conditions are understood and treated. It will help accelerate our ambitious immunoscience programs as we advance a rich pipeline of first- and best-in-class medicines across key therapeutic areas to address unmet patient needs."

Sanofi and Stanford Medicine will create a Joint Steering Committee to fund up to three programs a year. Sanofi will host an annual research forum for researchers from both organizations to further exchange ideas, share knowledge and perspectives on relevant scientific matters, and discuss collaborative research projects.

"Stanford Medicine is dedicated to advancing knowledge and discovery with the goal of improving our ability to predict, prevent and cure disease with the most precise approaches," said Lloyd Minor, MD, Dean of the Stanford School of Medicine. "The opportunity for long-term collaboration with our colleagues at Sanofi will allow us to explore together new frontiers in autoimmune diseases and inflammatory conditions."

The collaboration will begin with three "deep-dive" research projects:

Exploring cytokine crosstalk in type 2 inflammation, specifically examining the impact of Sanofi’s investigational molecules on excessive type 2 inflammation.
Decoding molecular drivers of effector and suppressor T cells in autoimmunity, to better understand the specific antigens that may cause type 1 diabetes.
Defining the mechanisms of immune-related adverse events with immune checkpoint inhibitor therapy – with a focus on pneumonitis and inflammatory arthritis – to explore the role of genomics and pathogenic cell identification.

EDITOR’S NOTE: This new initiative is the latest chapter in Sanofi’s long history of collaboration that includes the Sanofi Innovation Awards (iAwards), a multi-institutional academic partnership program designed to accelerate and innovative, early-stage, disease-relevant research towards the clinic. The iAwards represent one of the largest academic–industry collaborations run by a biopharma company, and have generated early research that has led to important scientific advancements in the field, including contributions to Sanofi’s own pipeline.

On May 6, 2021 Clarity Pharmaceuticals, a clinical stage radiopharmaceutical company focused on the treatment of serious disease, reported that the U.S. Patent and Trademark Office (USPTO) has granted the patent application for Clarity’s SAR-bisPSMA compound and its variants (Press release, Clarity Pharmaceuticals, MAY 6, 2021, View Source [SID1234579259]). The grant of the patent application bolsters Clarity’s strong Intellectual Property (IP) position on the Prostate Specific Membrane Antigen (PSMA) targeting agent for imaging and treatment of prostate cancer.

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SAR-bisPSMA is the result of Clarity’s optimisation of the well characterised PSMA targeting peptide, which has been in many thousands of patients to date, by combining two PSMA targeting peptides with Clarity’s proprietary SAR Technology and the theranostic isotopes of copper, copper-64 for imaging and copper-67 for therapy. The copper pairing enables diagnosis, staging and subsequent treatment of prostate cancers that express PSMA. The optimisation of the targeting peptide has resulted in superior targeting of, and retention in, tumours compared to a singular PSMA targeting peptide in preclinical models (Zia et al., 2019)1. The product was developed through the long-standing collaboration between Clarity and Professor Paul Donnelly at the University of Melbourne.

Clarity’s Executive Chairman, Dr Alan Taylor, commented: "The grant of the SAR-bisPSMA patent application is an example of the effectiveness of Clarity’s broad patent strategy around its SAR Technology, which allows the filing of additional patents with a wide range of new disease targeting agents."

The news from the USPTO come shortly after Clarity has completed the assignment of its key patent portfolio from the University of Melbourne, providing Clarity with the full rights and ownership of the patents.

"The grant of the SAR-bisPSMA patent application also comes soon after Clarity received a response from the U.S. Food and Drug Administration on its an Investigational New Drug (IND) application for a theranostic trial of 64Cu/67Cu SAR-bisPSMA in the U.S. (NCT04868604)2 that the study may proceed. With our diagnostic trial of 64Cu SAR-bisPSMA in previously diagnosed but yet untreated patients prior to surgical removal of the prostate in the start-up phase (NCT04839367)3, we are now ready to embark on two clinical trials of SAR-bisPSMA in the next few months," said Dr Taylor.

Prostate cancer is the second most common cancer diagnosed in men globally and the fifth leading cause of death worldwide4. The American Cancer Society estimates in 2021 there will be 248,530 new cases of prostate cancer in the U.S. and around 34,130 deaths from the disease5. For metastatic prostate cancer, the 5-year relative survival rate is 30%, indicating a high unmet need for early detection and better treatment options for mCRPC. Annually, there are around ~34,000 men in the U.S. who are diagnosed with mCRCP5, ~90% of whom have tumours which express PSMA6.

Clarity’s Executive Chairman, Dr Alan Taylor, commented, "The theranostic SAR-bisPSMA product has blockbuster market potential for prostate cancer. The product has ideal characteristics for a radiopharmaceutical and numerous advantages associated with the optimisation of the targeting peptide and using the copper pairing, including increased targeting and retention of the product in tumours, centralised manufacture, reaching more treatment sites and patients around the world, as well as diagnostic, dosimetry and therapeutic benefits. We are excited to progress the development of this proprietary asset into the clinic for the treatment of men with prostate cancer as part of our ultimate goal of improving treatment outcomes for kids and adults with cancer. We are pleased to have received the patent grant to reinforce Clarity’s IP position around our PSMA compounds, giving us confidence in the commercialisation process moving forward."

On May 5, 2021 Oncopeptides, a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, reported that the Company has completed patient enrollment in the phase 2 PORT study (Press release, Oncopeptides, MAY 5, 2021, View Source [SID1234646796]). The PORT study is an open-label, randomized, cross-over study which compares safety, tolerability and efficacy of peripheral or central intravenous administration of melflufen (INN melphalan flufenamide) in combination with dexamethasone in relapsed refractory multiple myeloma. Oncopeptides expects topline data in Q3 2021.
"I am very pleased that we have enrolled the final patient in the PORT study," said Klaas Bakker, MD, PhD and Chief Medical Officer at Oncopeptides. "The data could potentially provide a pathway for us to work with the U.S. Food and Drug Administration to include an additional mode of administration for PEPAXTO."

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"The continued development of melphalan flufenamide could potentially bring forward an additional therapeutic option to physicians and patients," said Joshua Richter, MD, Assistant Professor of Medicine, Hematology and Medical Oncology at The Tisch Cancer Institute at Mount Sinai and Site Director of Multiple Myeloma at the Blavatnik Family – Chelsea Medical Center at Mount Sinai, New York.

On May 5, 2021 OliX Pharmaceuticals Inc. (KOSDAQ: 226950) a leading developer of RNAi therapeutics, and PCI Biotech (OSE: PCIB), a cancer focused biopharmaceutical company with a unique intracellular delivery technology, reported an extensive research collaboration (Press release, PCI Biotech, MAY 5, 2021, View Source [SID1234585157]). OliX Pharmaceuticals and PCI Biotech will combine their know-how and technology platforms to explore synergies and further partnership on dermatology and other applications.

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The partnership is governed by a research collaboration agreement, under which the collaborators will perform an extensive evaluation of technology compatibility and synergy based on OLX104C (Androgenic Alopecia) preclinical studies. The companies will evaluate results achieved from this research collaboration to explore the potential for further development and partnership.

Commenting on the announcement, Dong Ki Lee, CEO of OliX Pharmaceuticals said: "We are very excited to collaborate with PCI Biotech. Combining our proprietary asiRNA platform with PCI’s leading fimaNAc delivery technology will significantly accelerate our efforts to bring our dermatological RNAi programs to the clinic. We also expect to expand the area of application to our cancer immunotherapy pipeline and mRNA vaccines developed by our subsidiary, mCureX, in the near future."

PCI Biotech’s CEO, Per Walday, added: "We are very pleased to initiate this exciting collaboration with OliX Pharmaceuticals. This collaboration builds on the strong potential of our intracellular delivery platform technology for nucleic acid based therapies, within a therapeutic field of good technological fit. Combining the leading technologies of OliX Pharmaceuticals and PCI Biotech may lead to new promising applications and we look forward to the collaboration."

On May 5, 2021 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the first quarter of 2021 (Press release, Sana Biotechnology, MAY 5, 2021, View Source [SID1234584006]).

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"We continue to make progress across our platforms and pipeline, targeting a broad set of diseases," said Steve Harr, Sana’s President and Chief Executive Officer. "In the first quarter, we bolstered our balance sheet, continued to build our capabilities, and moved forward our science. We recently presented scientific data at a medical conference for the first time, highlighting the potential of both our in vivo delivery platform and our ex vivo hypoimmune platform to make innovative CAR T therapies for cancer patients. We look forward to presenting more scientific data and progress updates from our various pipeline programs at conferences this year."

Recent Corporate Scientific Highlights

Presented proof of concept animal studies from the in vivo fusogen T cell and ex vivo hypoimmune allogeneic T cell programs at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021, highlighting the platforms’ ability to make potentially differentiated CAR T cells.
A single intravenous dose of targeted fusosomes enables specific delivery of a CD19 CAR transgene to CD8+ T cells, creating CAR T cells in vivo, that show a dose-dependent anti-tumor response regardless of prior T cell activation status.
Hypoimmunogenic CAR T cells show the ability to functionally evade the innate and adaptive immune system in allogeneic recipients and demonstrate tumor killing, potentially leading to universal CAR T cells that can persist without immunosuppression.
First Quarter 2021 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2021 were $981.9 million compared to $412.0 million as of December 31, 2020, an increase of $569.9 million. Sana successfully completed its initial public offering in February 2021 and issued 27.0 million shares of common stock, including 3.5 million shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, at a price of $25.00 per share, for net proceeds of $626.4 million.
Research and Development Expenses: Research and development expenses for the three months ended March 31, 2021, inclusive of non-cash expenses, were $168.9 million compared to $27.3 million for the three months ended March 31, 2020. The increase of $141.6 million was primarily due to non-cash expenses for the increase in the estimated fair value of the success payment liabilities in aggregate and contingent consideration of $115.7 million and $11.4 million, respectively. The increase was also due to personnel-related expenses related to increased headcount to expand Sana’s research and development capabilities, costs for preclinical studies, laboratory supplies, and facility costs. Research and development expenses include stock-based compensation of $2.5 million for the three months ended March 31, 2021 and $0.6 million for the three months ended March 31, 2020.
General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2021, inclusive of non-cash expenses, were $11.8 million compared to $6.0 million for the three months ended March 31, 2020. The increases of $5.8 million was primarily due to increased personnel-related expenses attributable to an increase in headcount to build our infrastructure, consulting and legal fees, insurance associated with being a public company, and facility costs. General and administrative expenses include stock-based compensation of $1.5 million for the three months ended March 31, 2021 and $0.1 million for the three months ended March 31, 2020.
Net Loss: Net loss for the three months ended March 31, 2021 was $180.6 million, or $1.52 per share, compared to $32.9 million, or $3.04 per share, for the three months ended March 31, 2020.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the three months ended March 31, 2021 was $48.9 million compared to $29.5 million for the three months March 31, 2020. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities excluding cash inflows from financing activities, cash outflows from business development activities, and the purchase of property and equipment.
Non-GAAP Research and Development Expenses: Non-GAAP research and development expenses for the three months ended March 31, 2021 were $41.9 million compared to $26.0 million for the three months ended March 31, 2020. Non-GAAP research and development expenses excludes one-time costs to acquire technology and non-cash expenses related to the change in the estimated fair value of contingent consideration and success payment liabilities.
Non-GAAP Net Loss: Non-GAAP net loss for the three months ended March 31, 2021 was $53.6 million, or $0.45 per share, compared to $31.6 million, or $2.92 per share, for the three months ended March 31, 2020. Non-GAAP net loss excludes one-time costs to acquire technology and non-cash expenses related to the change in the estimated fair value of contingent consideration and success payment liabilities.
A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under "Non-GAAP Financial Measures."