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."

On May 11, 2021Appia Bio, Inc., an early stage biotechnology company developing engineered allogeneic cell therapies from hematopoietic stem cells (HSCs) for cancer patients, reported its launch from stealth backed by $52 million Series A financing led by 8VC (Press release, Appia Bio, MAY 5, 2021, View Source [SID1234583854]). Other investors included Two Sigma Ventures, among others, and participation from seed investors Sherpa Healthcare Partners and Freeflow Ventures.

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Appia Bio is focused on discovering and developing off-the-shelf allogeneic cell therapies across a broad array of cancer indications, utilizing a scalable technology platform with the goal to increase access for patients. With its ACUA technology platform, Appia Bio leverages the biology of lymphocyte development with chimeric antigen receptor (CAR) and T-cell receptor (TCR) gene engineering to generate CAR-engineered invariant natural killer T (CAR-iNKT) cells from HSCs. The ACUA platform originated from groundbreaking research in the laboratory of Lili Yang, PhD, associate professor at University of California, Los Angeles (UCLA), and her collaborations with the Company’s other scientific founders.

Proceeds from the financing will support the advancement of Appia Bio’s pipeline of allogeneic CAR-iNKT cell therapy candidates into the clinic. As part of the Series A financing, Francisco Gimenez, PhD, partner, and David Moskowitz, PhD, principal, at 8VC, join the Company’s board of directors, and David Baltimore, PhD, of the California Institute of Technology (Caltech) was elected chairman of the Board.

"There is tremendous potential to be explored in allogeneic cell therapies. The Board and I look forward to working with this stellar team to create transformative cell therapies that will help patients and the physicians who treat them," said Dr. Baltimore.

"We are grateful to our new and existing investors for their commitment to bring off-the-shelf allogeneic cell therapies to more patients," said JJ Kang, PhD, co-founder and chief executive officer (CEO) of Appia Bio. "This financing will position us to rapidly build out our CAR-iNKT pipeline and establish the clinical potential of our ACUA platform. We are excited to work with an outstanding Scientific Advisory Board that brings together world-class expertise and experience across the many fields necessary to develop cell therapies."

Appia Bio announced the establishment of a Scientific Advisory Board with leaders in immunology, immuno-oncology, and cell therapy engineering. These experts join Appia Bio’s scientific founders in advising the Company’s research and development.

Mitchell Kronenberg, PhD, president and CSO, La Jolla Institute for Immunology

Antoni Ribas, MD, PhD, professor of medicine, UCLA, and director of the Tumor Immunology Program at the Jonsson Comprehensive Cancer Center

Margo Roberts, PhD, former CSO, Kite Pharma and Lyell Immunopharma

"We are thrilled to partner with Appia Bio as it typifies the innovative, founder-driven start-ups we love to empower," said Dr. Gimenez. "Appia unifies compelling biology of iNKT cells with the power of CARs. This is only possible because of their fundamental innovations in manufacturing and engineering these cells."

The Company was founded in 2020 in Los Angeles, California, by a team of experts and industry leaders with a proven track record of cell therapy development and company building.

David Baltimore, PhD, Nobel Laureate, president emeritus and distinguished professor of biology, Caltech

JJ Kang, PhD, CEO, Appia Bio; former partner, The Column Group

Edmund Kim, PhD, chief operating officer of Appia Bio; former vice president of corporate development, Kite Pharma

Pin Wang, PhD, professor of chemical engineering and materials science and biomedical engineering, University of Southern California (USC)

Jeff Wiezorek, MD, chief medical officer, Appia Bio; former head of cell therapy development, Kite Pharma

Lili Yang, PhD, associate professor of microbiology, immunology, and molecular genetics, UCLA

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United Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On May 5, 2021 Race Oncology Limited (ASX: RAC) reported that it has received binding commitments to raise $5.4m (before associated costs) in an oversubscribed equity placement to new and existing institutional and sophisticated investors (Placement), and the commencement of a bonus issue of options to existing eligible shareholders of Race (Bonus Option Issue) (Press release, Race Oncology, MAY 5, 2021, View Source [SID1234580021]).

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"This new funding supports efforts to deliver outsized returns to shareholders via our Three Pillar strategy, where we are investigating Bisantrene as both a potential precision oncology agent, and as a heart-friendlier chemotherapeutic. We appreciate the amount of interest shown in both Race and Bisantrene through this process and thank all those who participated in the Placement."

Race’s CEO and Managing Director, Phil Lynch
"We thank our new and existing shareholders for their continuing support. We believe that this raise will accelerate our plans and rewards our loyal shareholders. Race remains efficient with our capital usage and will always be focused on achieving the best outcome for our shareholders and the patients who may be helped by Bisantrene."

Dr Daniel Tillett, Race’s CSO and Executive Director
About the Placement
Race will raise $5.4m (before associated costs) under the Placement by the issue of approximately 1.8m new fully paid ordinary shares (New Shares) at an issue price of $3.00 per share. The issue price represents a 2.3% discount to the last traded price and an 7.8% discount to the 10-day volume weighted average price of $3.25.

Participants in the Placement will receive 1 free attaching option (exercisable at $4.50 and expiring 16 May 2022) (Placement Option) for every 20 New Shares subscribed for and issued under the Placement. The Placement, including the Placement Options, is not subject to shareholder approval.

The cornerstone investor to the Placement was existing Race shareholder, Merchant Opportunities Fund. Merchant Opportunities Fund is a boutique fund with a number of long-term, strategic investments in the Australian biotechnology industry. Race accepted bids above $5m to cover all costs in managing the Placement leaving approximately $5m for planned activities.

Merchant Capital Partners Pty Ltd and MST Financial Services Pty Ltd acted as Joint Lead Managers and bookrunners to the Placement. IR Department provided investor relations support.

The New Shares and Placement Options are expected to be allotted and issued by 14 May 2021 and New Shares will rank equally with the existing ordinary shares on issue. The New Shares and Placement Options will be issued pursuant to the Company’s existing placement capacity under ASX listing rule 7.1.

Use of Funds
The Placement provides Race with additional capital to be primarily deployed toward its Three Pillar strategy and supporting plans:

Pillar 1: Phase 1 dose escalation trial to determine optimal FTO-targeted Phase II dosage
Pillar 2: Preclinical study to determine the molecular mechanism of action of Bisantrene’s heart safety
Pillar 3: Initiation of studies in Europe and the United States to support extramedullary Acute Myeloid Leukaemia clinical trials
Corporate: scaled manufacturing of Bisantrene to support Phase II/III trials
About the Bonus Option Issue
Race is also pleased to announce a pro-rata non-renounceable Bonus Option Issue (exercisable at $4.50 and expiring 16 May 2022) (Bonus Options) to existing eligible shareholders on the basis of 1 Bonus Option for every 20 shares held on the record date.

The primary purpose of the Bonus Option Issue is to reward loyal shareholders in Race, while efficiently growing the Company.

The Bonus Option Issue is being extended to eligible shareholders who have a registered address in Australia or New Zealand at 7:00 pm AEST on 13 May 2021.

The Bonus Options and the Placement Options will be in the same class and will not be listed. The details of the offer are contained in the Bonus Option Prospectus below.