On November 8, 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 third quarter of 2021 (Press release, Sana Biotechnology, NOV 8, 2021, View Source [SID1234594793]).

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"We continue to make progress in building the company, progressing our pipeline, and growing our people and capabilities," said Steve Harr, Sana’s President and Chief Executive Officer. "The recent licensing of genome editing technology to enable multiple pipeline programs is an example of our continued focus on augmenting our innovative capacity. As our pipeline progresses, we look forward to presenting data from our in vivo CAR T and ex vivo allogeneic CAR T cell programs at the upcoming 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in December."

Recent Corporate Highlights

Announced an agreement for non-exclusive commercial rights to Beam’s CRISPR Cas12b nuclease system for certain ex vivo engineered cell therapy programs. Engineering cells for therapeutic applications requires technologies for precise editing of their genome sequence. We plan to use the technology with certain product candidates, including many of our allogeneic CAR T and pluripotent stem cell programs.
Third Quarter 2021 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of September 30, 2021 were $866.1 million compared to $412.0 million as of December 31, 2020, an increase of $454.1 million. The increase was primarily driven by net proceeds of $626.4 million received in Sana’s initial public offering in February 2021, partially offset by cash used in operations of $141.0 million and cash used for the purchase of property and equipment of $24.7 million.

Research and Development Expenses: For the three and nine months ended September 30, 2021, research and development expense, inclusive of non-cash expenses, was $53.2 million and $140.2 million, respectively, compared to $40.1 million and $96.5 million, respectively, for the same periods in 2020. The increases of $13.1 million and $43.6 million, respectively, for the three and nine months ended September 30, 2021 were due to an increase in personnel expenses related to increased headcount to expand Sana’s research and development capabilities, costs for laboratory supplies, costs for preclinical studies and external manufacturing, and facility costs. Research and development expenses include non-cash stock-based compensation of $4.1 million and $9.9 million, respectively, for the three and nine months ended September 30, 2021 and $1.0 million and $2.6 million, respectively, for the same periods in 2020.

Research and Development Related Success Payments and Contingent Consideration: For the three and nine months ended September 30, 2021, we recognized non-cash expenses of $16.8 million and $67.8 million, respectively, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration, compared to $4.5 million and $57.3 million, respectively, for the same periods in 2020.

General and Administrative Expenses: General and administrative expenses for the three and nine months ended September 30, 2021, inclusive of non-cash expenses, were $13.4 million and $37.7 million, respectively, compared to $7.1 million and $19.1 million, respectively, for the same periods in 2020. The increases of $6.3 million and $18.6 million, respectively, in the three and nine months ended September 30, 2021 were primarily due to increased personnel-related expenses attributable to an increase in headcount to build our infrastructure, legal fees to support our patent portfolio and license arrangements, insurance associated with being a public company, consulting fees, and facility costs. General and administrative expenses include stock-based compensation of $1.9 million and $5.2 million, respectively, for the three and nine months ended September 30, 2021 and $0.2 million and $0.5 million, respectively, for the same periods in 2020.

Net Loss: Net loss for the three and nine months ended September 30, 2021 were $83.3 million, or $0.46 per share, and $245.2 million, or $1.53 per share, respectively, compared to $51.5 million, or $3.76 per share, and $172.1 million, or $14.05 per share, respectively, for the same periods in 2020.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the nine months ended September 30, 2021 was $146.4 million compared to $87.2 million for the nine months September 30, 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 and nine months ended September 30, 2021 were $53.2 million and $140.1 million, respectively, compared to $31.6 million and $86.5 million, respectively, for the same periods in 2020. Non-GAAP research and development expenses excludes one-time costs to acquire technology.

Non-GAAP Net Loss: Non-GAAP net loss for the three and nine months ended September 30, 2021 was $66.5 million, or $0.37 per share, and $177.4 million, or $1.11 per share, respectively, compared to $38.5 million, or $2.81 per share, and $104.9 million, or $8.56 per share, respectively, for the same periods in 2020. Non-GAAP net loss exclude 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."

On November 8, 2021 Leadingtac Bio, a company in Zhangjiang Science City announced the completion of a Pre-A round of financing led by Yuan Sheng Venture Capital, with additional investment from long-time shareholder ZJ LEADING VC (Press release, Leadingtac Pharmaceutical, NOV 8, 2021, View Source [SID1234640717]). This funding round will be used primarily to advance the company’s first Protein Degrader pipeline for autoimmune disease treatment to the clinical research stage and to develop follow-on programs and pipelines, including oncology and other autoimmune diseases. With the previous angel round, Leadingtac has raised nearly 100 million RMB in cumulative early-stage financing.

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Leadingtac Bio was founded by Dr. Yan Feng in September 2019 in Zhangjiang Science Park. The company’s R&D direction is dedicated to the development of target protein degradation drugs and focuses on disease areas such as autoimmunity and oncology to address unmet clinical needs.

The target protein degradation drug is a bifunctional small molecule compound, a ligand that binds the target protein at one end and E3 ubiquitin ligase at the other. A Linker connects the ligands to form a complex that draws the target protein closer to the E3 ligase and promotes the ubiquitination of the target protein, which is then degraded through the ubiquitin-proteasome pathway to remove the disease-causing target protein. Targeted protein degradation drugs are characterized by high drug-forming ability, overcoming drug resistance, small dosage, low toxicity, and drug resistance. Therefore, this research direction has received close attention from domestic and international pharmaceutical companies and capital markets.

Since its inception, the company has taken the mission of "leading innovation, Thailand’s people", based on the source of innovation in new drug development, looking for new targets, using protein degradation technology to develop new small molecule drugs that are currently not drug-ready targets, to provide maximum therapeutic value for patients. As a result, we have developed a series of lead compounds with fully independent intellectual property rights based on our Nano-SPUD platform for specific protein ubiquitination and degradation drug discovery. The company’s fastest progressing project is the LT001 project for autoimmune diseases. The project has obtained expected results from a series of in vitro and in vivo experiments, such as degradation activity, pharmacokinetics, and efficacy, and has already carried out IND-enabling studies, which are expected to be submitted to IND at the end of 2022 and enter clinical trials in 2023. In addition, Leadingtac is also actively developing several pipeline projects.

Dr. Yan Feng, the founder of Leadingtac Bio, said, "As a significant direction for future drug development, the development of targeted protein degradation drugs (Protein Degrader) has received much attention from academia, the pharmaceutical industry, and the capital market in the past few years. As a newcomer in this field, Leadingtac has developed its R&D technology platform and pipeline within two years of its efforts. One of the pipelines will enter the clinical filing stage soon. In this financing round, Leadingtac was honored to receive the approval and lead investment from Yuan Sheng Venture Capital, and ZJ LEADING VC also decided to continue to invest. We are very grateful to our new and old investors for their recognition and support. This financing round will help to rapidly advance the clinical filing of Leadingtac’s first pipeline and also provide a strong guarantee for developing several subsequent projects, high-end talent team building, and site expansion. I believe that with the help of our investors and the efforts of the team at Leadingtac, we will be able to maximize our potential for new drug development and make greater contributions to the pharmaceutical field!"

Jay Chen, Founding Partner of Yuan Sheng Ventures, said, "We are pleased to invest in Leadingtac as the lead investor in this round. As a newcomer in the field of Protein Degrader, Leadingtac Bio has established a more mature technology platform, the first product has also started the filing of IND, and the subsequent product pipeline is various and has great potential for development. As a result, we believe Leadingtac Bio will have great international competitiveness in the future."

Yu Xiaoyong, the founding managing partner of ZJ LEADING VC, said, "We congratulate Leadingtac on the completion of this financing round. As the round angel investor of Leadingtac, we had the honor to work with Dr. Yan Feng two years ago to help Leadingtac plow through the new drug development track of protein degradation. Over the past two years, we have witnessed the continuous growth of Leadingtac’s team and the company together and have gained a deep understanding of the company’s continuous innovation powerhouse, which is why we have sufficient confidence in Leadingtac’s future development, and that is why we continue to invest in this round. We believe that after this round of financing, Leadingtac Bio will continue to achieve good results in the advancement of clinical filing projects, the layout of pipeline projects, and the reinforcement of the team."

On November 8, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported that Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector, will participate in a fireside chat at the Stifel 2021 Virtual Healthcare Conference on Monday, November 15, 2021, at 2:00 p.m. ET (Press release, Alector, NOV 8, 2021, View Source [SID1234594697]).

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A live webcast of the fireside chat will be available on the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 90 days following the event.

On November 8, 2021 Celsion Corporation (NASDAQ: CLSN), a clinical-stage development company focused on DNA-based immunotherapy and next-generation vaccines, reported that Khursheed Anwer, Ph.D., executive vice president and chief science officer, will be making a presentation on the company’s GEN-1 interleukin 12 (IL-12) immunotherapy program at the Cytokine-Based Cancer Immunotherapies Summit being held in Boston on November 30 to December 2, 2021 (Press release, Celsion, NOV 8, 2021, View Source [SID1234594713]). Dr. Answer will also be participating in two panel discussions.

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In his presentation, Dr. Anwer will be discussing how local delivery of IL-12 without significant systemic toxicity is feasible with a non-viral gene therapy approach that involves administration of an IL-12 plasmid with a synthetic DNA delivery system (GEN-1). Dr. Anwer will also be discussing how weekly intraperitoneal administration of GEN-1 yields durable increases in IL-12 and IFN-g, and why repeated weekly administration of GEN-1 in combination with standard chemotherapy remodels the tumor immune environment to favor immune stimulation over immune suppression.

"Dr. Anwer’s presentation and panel discussions will highlight the strength of our GEN-1 immunotherapy program to some of the nation’s leading immunologists," said Michael H. Tardugno, chairman, president and chief executive officer of Celsion. "We look forward to interacting with attendees at one of the first in-person medical conferences we will be attending since the COVID-19 pandemic began."

Dr. Anwer will participate in a group panel discussion on November 30 at 7:40 a.m. Eastern time titled, "What Do We Know & Where Do We Want to Go?" and in another panel discussion on December 1 at 11:30 a.m. Eastern time titled "Side Effects – Mitigating Against Hypotension + Fever With Immune-Stimulating Agents (NK Cell Engagers, PD-1s, Cytokines, T-Cell Engagers) = Cytokine Release Syndrome (CRS)?" Dr. Anwer’s presentation, titled "A Non-Viral Gene Therapy Approach to IL-12 Delivery for The Treatment of Cancer," will be delivered on December 1 at 8:30 a.m. Eastern time.

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system that enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anticancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The company previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION 1 Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer. GEN-1 in combination with neoadjuvant chemotherapy is the subject of the ongoing Phase II OVATION 2 Study in subjects with advanced-stage ovarian cancer (Stage III/IV), with enrollment now exceeding 75% and full enrollment targeted by the first half of 2022.

On November 8, 2021 Doloromics has reported the appointment of Dr. Chris Flores as their new President and Chief Research and Development Officer (Press release, Lifescience Newswire, NOV 8, 2021, View Source [SID1234594729]). In this role, Dr. Flores will be responsible for leading the Doloromics portfolio of drug discovery and development programs, two of which are scheduled to launch in early 2022, while building relationships with strategic partners and collaborators in support of these efforts.

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"After an extensive and diverse experience in the Pharmaceutical and Consumer sectors of Johnson & Johnson, I’m both thrilled and privileged to commit the full measure of my expertise and passion in executing the core mission of Doloromics, to eradicate pain in patients who remain tragically underserved by current standards of care." — Chris Flores, PhD.

Dr. Flores will complement the Doloromics leadership team after a successful, 19-year career at Johnson & Johnson, where he held several executive positions, including Vice President of Neuroscience, La Jolla R&D Site Head, Pain Franchise Strategy Leader, Head of Pain Discovery and Global External Innovation Head of Discovery Sciences within Janssen Research & Development as well as Head of Emerging Science and Innovation Strategy for J&J Consumer Companies.