On February 24, 2021 Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA therapeutics (oRNAs), reported the completion of its $80 million Series A financing (Press release, OrnaTherapeutics, FEB 24, 2021, View Source [SID1234626172]). Founded on groundbreaking research from the Massachusetts Institute of Technology (MIT) and built by MPM Capital, Orna utilizes elegant molecular design and delivery technologies to overcome the limitations of linear mRNA therapeutics and unlock RNA’s full therapeutic potential to treat a wide range of diseases including cancer, autoimmune, and genetic disorders. Led by Thomas M. Barnes, Ph.D., as Chief Executive Officer, Orna’s team of skilled scientists, molecular engineers, and industry veterans leverage their broadly applicable oRNA platform to dramatically change and improve current approaches to treatment with the potential to make a difference in the lives of patients. The financing was co-led by MPM Capital, Taiho Ventures, and F2 Ventures, with participation from leading strategic investors: Kite, a Gilead Company, Bristol Myers Squibb, Astellas Venture Management, Novartis Institutes for Biomedical Research, and the PAGS Group.

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Orna’s engineered circular RNAs have several key advantages, including superior protein expression, simpler and more cost-effective manufacturing, and improved delivery. Unlike other RNA therapies, oRNA does not require the addition of cap or tail structures and does not require the use of modified nucleotides to address innate immune responses. In addition, oRNAs are designed to drive unparalleled protein production while resisting degradation inside the body.

"Our elegant solution to the circular RNA engineering problem has allowed us to reveal that oRNA is simply the better format for long coding RNA," said Dr. Barnes. "Furthermore, our initial combination of oRNAs with technology to deliver them to immune cells has paved the way to create groundbreaking new therapies to fundamentally change the way we treat cancer and autoimmune diseases."

The company was founded by (Robert) Alex Wesselhoeft, Ph.D., Raffaella Squilloni, M.B.A. and Professor Dan Anderson, Ph.D. (MIT), and was seeded and incubated by MPM Capital, with Shinichiro Fuse, Ph.D. serving as the interim CEO, and Brian Goodman, Ph.D. as head of Business Development. "We believe that oRNA is the future, and Orna has built the right team to execute on this vision," said Ansbert Gadicke, M.D., co-founder and Managing Director, MPM Capital, Board Chair of Orna Therapeutics. "Orna’s elegant and meticulously calculated approach has the potential to forever change the field of mRNA therapeutics."

The potential applications of Orna’s technology are far reaching. In particular, Orna believes it can apply its oRNA technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (in situ CAR therapy). The Series A financing will enable Orna to bring its initial programs to IND enablement, while continuing to advance its robust platform and build the capabilities to support Orna’s mission in its new office and laboratory space located in Cambridge, Massachusetts. Orna is supported by an experienced and seasoned Board of Directors and scientific advisors, including:

Daniel Anderson, Ph.D., Professor, MIT
Sakae Asanuma, CFA, MBA, President, Taiho Ventures
Thomas M. Barnes, Ph.D., CEO, Orna Therapeutics
Shinichiro Fuse, Ph.D., Managing Director, MPM Capital
Ansbert Gadicke, M.D. (chair), co-founder and Managing Director, MPM Capital
Morana Jovan-Embiricos, Managing Partner, F2 Ventures
Francesco Marincola, M.D., SVP and Global Head of Cell Therapy Research of Kite

On February 24, 2021 Verity Pharmaceuticals reported that it has received a notice of compliance with conditions (NOC/c) from Health Canada for the VERITY-BCG (Bacillus Calmette-Guérin [BCG]: Strain Russian BCG-I) (Press release, veritypharma, FEB 24, 2021, View Source [SID1234605586]). This approval is welcome news for patients and health care providers who have endured a Canadian and global shortage of this important bladder cancer drug.

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Bacillus Calmette-Guérin (BCG) is a proven immunotherapy of choice for bladder cancer. It is used as first-line intravesical therapy following tumor resection of non-muscle-invasive bladder cancer. Primary producers of BCG announced shortages within the last decade, leading to a worldwide scarcity of the treatment. This new source of BCG from Verity Pharma is expected to be available by mid 2021.

"Canada’s multi-year BCG drug shortage will now be a thing of the past with this approval," said Howard Glase, Chief Executive Officer, Verity Pharma. "We know these drug shortages were leading to rationing and dose sparing of this crucial bladder cancer drug and patients had delayed or even cancelled treatments. Canadian patients will now have full access to this life-saving therapy."

According to an article by the National Centre for Biotechnology Information, "physicians treating patients affected by non-muscle invasive bladder cancer (NMIBC) have been in shock during the last six years since manufacturing restrictions on the production of the first-option medicine, Mycobacterium bovis Bacillus Calmette-Guérin (BCG), have resulted in worldwide shortages."i

Bladder Cancer Canada states that bladder cancer is the 5th most common cancer in Canada and almost 12,000 people will be diagnosed with bladder cancer this year alone.

Verity has partnered with Serum Institute of India, which manufactures BCG. Serum Institute is the world’s largest manufacturer of vaccines.

"Verity is proud to continue bringing critical drugs to Canadians. VERITY-BCG is an example of our expertise in supplying life-saving therapeutic options to help fulfill unmet patient needs," said Glase.

About VERITY-BCG

VERITY-BCG is an adjuvant therapy after transurethral resection (TUR) of a primary or relapsing superficial papillary urothelial cell carcinoma of the bladder stage Ta (grade 2 or 3) or T1 (grade 1, 2, or 3), without concomitant carcinoma in situ. It is only recommended for stage Ta grade 1 papillary tumors, when there is judged to be a high risk (>50%) of tumor recurrence.

More information is available here: View Source

On February 24, 2021 Rapha Capital Management, LLC (View Source), an investment management firm located in Miami, Florida, reported that on February 17, 2021, its portfolio company, NexImmune, Inc. ("NexImmune"), closed an upsized initial public offering raising gross proceeds, before deducting underwriting discounts and commissions and offering expenses, of approximately $126.5 million (Press release, Ponce Therapeutics, FEB 24, 2021, View Source;utm_medium=rss&utm_campaign=rapha-capital-management-announces-successful-initial-public-offering-of-portfolio-company-neximmune-inc [SID1234578720]). The gross proceeds included the exercise in full by the underwriters of their option to purchase additional shares. NexImmune’s shares are traded on the Nasdaq Global Market under the symbol "NEXI". Rapha Capital Investment II, LLC ("RCI II") (an entity managed by Rapha Capital), participated in NexImmune’s Series A financing in July, 2018 and Series A-2 financing in January, 2019, investing a total of $1.8 million.

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Rapha Capital Management is an investment management firm focused on managing strategic investments in early stage, non-public biotechnology companies, through special purpose investment vehicles ("SPIVs"). Rapha Capital was founded by its President, Kevin Slawin, M.D., a successful and experienced oncologic and robotic surgeon. After leaving practice, Dr. Slawin has been serving as a biotech consultant, investor, and founder, focusing on disruptive technologies in oncology, T cells and immunotherapy, and other breakthrough healthcare technologies. He is the founder of Bellicum Pharmaceuticals, Inc. ("Bellicum"), a publicly traded company listed on NASDAQ, leading Bellicum to a successful $161 million IPO in December, 2014. He also plays a guiding role in several of the investments managed by Rapha Capital in certain companies, serving as a board member at 3DBio Therapeutics, Inc. (View Source), FIZE Medical, Inc. (www.fizemedical.com), and Demeetra AgBio, Inc. (www.demeetra.com). He served as a board member and interim CEO of portfolio company AsclepiX Therapeutics, Inc. (www.asclepix.com) in 2020, engineering their $35 million Series A financing led by Perceptive Xontogeny Venture Fund in mid 2020. Rapha Capital Management manages thirteen legacy SPIVs, Rapha Capital Investment I – XIII. Rapha Capital Management will be offering alternative asset management services to its inaugural venture capital fund, launching at the end of 1Q21, which will be the vehicle for all future investments managed by Rapha Capital Management.

"We recognized the transformative potential of immunotherapy early on, founding Bellicum Pharmaceuticals, one of the first cellular immunotherapy companies in 2004, and have been an expert in identifying other potentially disruptive technologies, since 2017, when Rapha Capital Management was founded," commented Kevin Slawin, MD. "RCI II’s investment in NexImmune is another example of our commitment to use our unique abilities to identify at the earliest stage, those companies that we believe will go on to profoundly improve peoples’ lives while building great value, from a sea of seemingly indistinguishable, less compelling, opportunities," he added.

On February 24, 2021 Cerus Corporation (Nasdaq: CERS) and Shandong Zhongbaokang Medical Implements Co. Ltd. (ZBK) reported that they are forming a joint venture (JV) with the intent to develop, obtain regulatory approval for, manufacture and commercialize the INTERCEPT Blood System for platelets and red blood cells in China (Press release, Cerus, FEB 24, 2021, View Source [SID1234575789]). The JV, which will be named "Cerus ZBK Biomedical" (CEZB), will be headquartered in Zibo, Shandong Province in eastern China.

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"We are honored to join forces with ZBK in China, furthering our mission to make INTERCEPT the standard of care for patients globally," said Pascal Maillard, Cerus’ vice president of commercial operations for the Asia Pacific region. "We believe that ZBK’s experience with local clinical and regulatory requirements, ISO-certified manufacturing infrastructure, and existing sales channels will facilitate accelerated and broad access to INTERCEPT across China’s transfusion medicine community."

China represents one of the largest potential blood transfusion markets globally for INTERCEPT, with red blood cell collections in excess of 23 million unit-equivalents each year, and an estimated production of 1.8 million platelet doses per year. The healthcare system in China continues to grow at a rapid pace, enabling expanded access to blood components expected to benefit from pathogen inactivation.

"We are delighted to collaborate with Cerus to work to bring INTERCEPT to China as the market for platelets continues to grow rapidly," said Mr. Xu Junfeng, chief executive officer of ZBK. "INTERCEPT-treated platelets would address an unmet clinical need in the blood transfusion market in China and would offer an important new, prospective measure of safety against known and emerging pathogens during this new era of pandemic preparedness planning."

ZBK is a subsidiary of the Taibao Group and a leading developer, manufacturer and marketer of blood transfusion, blood safety, and infusion products in China. ZBK markets and sells its products through its own sales force network to over 280 blood centers nationwide.

Under the terms of the JV agreement, Cerus and ZBK are the sole shareholders in CEZB, with Cerus owning a majority (51%) of the entity. Cerus anticipates that it will consolidate the results of CEZB in its consolidated financial statements. Additionally, Cerus will contribute an exclusive license to the INTERCEPT Blood System for platelets and red blood cells for CEZB to market across China. ZBK will contribute its significant local expertise to help perform necessary clinical studies and seek regulatory approval. Ultimately it is expected that ZBK will establish local manufacturing and leverage its commercial expertise and presence in the region.

On February 24, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported that Chief Executive Officer, Gil Beyen, will present at the following conferences in the month of March and participate in select one-on-one investor meetings alongside members of the senior management (Press release, ERYtech Pharma, FEB 24, 2021, View Source [SID1234575739]).

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Cowen and Company – 41st Annual Health Care Conference – March 1-4, 2021

Gil Beyen, Chief Executive Officer, will participate in the GI Oncology and Pancreatic Cancer Panel Discussion on March 2, 2021 at 9:50am ET and management will conduct one-on-one meetings with investors during the conference.

The GI Oncology and Pancrearic Cancer Panel will stream live and a replay will be available on Cowen’s Conference Site: View Source

H.C. Wainwright – Global Life Sciences Conference, March 9-10, 2021

A pre-recorded corporate presentation will be accessible to conference attendees starting on Tuesday, March 9th 2021 at 7am ET to 90 days after the event. Management will conduct one-on-one meetings with investors during the conference.

A link to the HCW webcast will be accessible via ERYTECH’s website at View Source

If you are interested in arranging a one-on-one meeting request please contact your bank conference representative or contact Corey Davis at LifeSciAdvisors.