Mereo BioPharma Reports Full Year 2020 Financial Results and Recent Highlights

On March 31, 2021 Mereo BioPharma Group plc (NASDAQ: MREO) ("Mereo" or the "Company"), a clinical-stage biopharmaceutical company focused on oncology and rare diseases, reported financial results and for the year ended December 31, 2020 and provided an update on recent corporate highlights (Press release, Mereo BioPharma, MAR 31, 2021, View Source [SID1234577489]).

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"Despite the challenging landscape presented by the ongoing pandemic, this past year has been one of continued execution for Mereo, and I believe that 2020 was a highly successful and exciting year for the Company," said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. "We were able to further strengthen our partnering portfolio with licensing agreements for setrusumab and navicixizumab, with significant milestone payments tied to each deal. Our internal pipeline has continued to progress with etigilimab currently in a Phase 1b/2 basket study and alvelestat in a Phase 2 POC study for patients with AATD as well as a Phase 1 study in patients with COVID-19. Since the beginning of 2020, we successfully raised a total of $183 million (£138 million) through a combination of private placements, convertible loan notes and most recently a public offering in February 2021. The proceeds from these financing events, coupled with the upfront payments under the setrusumab and navicixizumab licensing agreements, will fund the continued advancement of our clinical programs and allow us to continue focusing on execution of our clinical and operational development strategies. As we look toward 2021, I believe that the Company is well positioned to deliver on multiple milestones and build upon the momentum we generated in 2020 as we continue to advance to our goal of becoming a leading biopharmaceutical company developing innovative therapeutics to improve outcomes for patients with rare diseases and select oncology indications."

Recent Product Highlights and Developments

Etigilimab (OMP-313M32)

Initiated Phase 1b/2 basket study in combination with an anti-PD-1 in a range of tumor types
Initial data expected second half 2021
Alvelestat (MPH-966)

Ongoing Phase 2 trial in 165 patients with AATD
Data expected in late 2021
Initiated Phase 1 study for the treatment of COVID-19 – data expected second half 2021
Setrusumab (BPS-804)

Rare pediatric disease designation in September 2020
Announced partnership with Ultragenyx for the development of setrusumab for the treatment of patients with OI in December 2020
Navicixizumab (OMP-305B83)

In January 2020 completed a global license agreement with OncXerna Therapeutics (formerly Oncologie, Inc.) for the further development and commercialization of navicixizumab.
Corporate Updates

Strengthened Management team

John Lewicki, PhD appointed Chief Scientific Officer, and Ann Kapoun, PhD appointed SVP Translational R&D, June 2020
Christine Fox appointed Chief Financial Officer, and Heidi Petersen appointed SVP Regulatory Affairs, October 2020
Suba Krishnan, M.D. appointed Senior Vice President of Clinical Development, November 2020
Delisted From AIM

Officially delisted from the AIM market of the London Stock Exchange on December 18, 2020
The Company’s American Depositary Shares ("ADSs") remain listed, and are only tradeable on Nasdaq
Upcoming Events

Needham Healthcare Conference, April 12-15, 2021
Jefferies Healthcare Conference, June 1-4, 2021
Full Year 2020 Financial Results

Full year 2020 research and development expenses were £16.3 million, compared to £23.6 million in 2019. R&D expenses relating to setrusumab decreased by £6.0 million, or 44%. The decrease was driven primarily by the completion of the adult Phase 2b study which reported top-line data in November 2019, with a further update in January 2020. Following the licensing and collaboration agreement with Ultragenyx, future ongoing development costs for setrusumab are expected to decrease significantly. R&D expenses relating to alvelestat remained consistent, reflecting the ongoing Phase 2 proof-of-concept study. R&D expenses relating to leflutrozole decreased by £1.0 million, or 88%, due to the completion of the Phase 2b study in 2019 and limited activity in 2020 following the completion of the study. Similarly, there were no ongoing studies for acumapimod in 2020 and this resulted in a decrease in R&D expenses for acumapimod of £0.3 million, or 72%. Partially offsetting the decrease, R&D expenses relating to etigilimab increased by £0.3 million, or 34%. The increase was driven primarily by the costs associated with preparing for the open label Phase 1b/2 basket study in combination with an anti-PD-1 in a range of tumor types. We expect the costs related to the etigilimab program to increase significantly in 2021.

Administrative expenses increased by £5.3 million, or 33%, from £15.9 million in 2019 to £21.2 million in 2020. The increase was primarily due to incremental legal and professional fees associated with various transactions during the year. Professional and legal fees increased from £1.7 million to £6.9 million in 2019 and 2020, respectively. The increase reflects transaction costs associated with the June 2020 Private Placement and the cancellation of admission of our ordinary shares to trading on the AIM market of London Stock Exchange in December 2020, along with higher costs associated with the Nasdaq listing and managing a larger business in two jurisdictions following the acquisition of Mereo BioPharma 5, partially offset by intellectual property related costs as a result of lower activity associated with setrusumab. Employee-related costs increased by £1.5 million to £7.3 million in 2020 primarily due to the expansion of our management team in 2020 compared to 2019. Premises-related costs increased by £1.7 million in 2020 primarily due to transaction costs associated with renegotiation of our office lease in Redwood City. This was partially offset by a gain on lease modification of £0.9 million. Offsetting these increases were lower travel-related costs, which decreased by £0.5 million from 2019 due to COVID-19 travel restrictions.

Net loss attributable to equity holders for the year 2020 was £163.6 million, compared to a net loss of £34.8 million in 2019, reflecting an operating loss of £37.6 million, a loss of £109.8 million due to changes in the fair value of financial instruments and a £10.9 million loss on disposal of intangible assets.

Total ordinary shares outstanding at December 31, 2020 were approximately 339 million shares. Total ADSs outstanding at December 31, 2020 were approximately 67.7 million, with each ADS representing five ordinary shares of the Company.

Cash and short-term deposits totaled £23.5 million as of December 31, 2020. Mereo anticipates that its current cash and short-term deposits, which includes the upfront payment received under the collaboration and license agreement with Ultragenyx and our recently completed public offering in February 2021, will extend the Company’s runway into 2024.

Edison Oncology Partners DNA-damage Response Inhibitor Program with Rakovina Therapeutics

On March 31, 2021 Edison Oncology Holding Corp. ("Edison Oncology"), a company established to develop and commercialize new therapies targeting the fight against cancer, reported that it has transferred certain worldwide rights to novel DNA-damage response (DDR) inhibitors, including its EO2000 series PARP Inhibitor Program Technology, to Rakovina Therapeutics Inc. ("Rakovina", TSX-V: RKV) (Press release, Edison Oncology, MAR 31, 2021, View Source [SID1234577472]).

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In exchange for transferring these rights, Edison Oncology has been issued common shares representing approximately 43% of the outstanding shares of Rakovina. Edison Oncology’s ownership in Rakovina is held by NewGen Therapeutics, Inc. a wholly-owned subsidiary of Edison Oncology.

"We are pleased to work with the Rakovina Therapeutics team to establish this new company that will seek to develop new cancer treatments based on novel DNA-damage response technologies", said Mr. Jeffrey Bacha, Edison Oncology’s chief executive officer. "Edison Oncology and its shareholders will benefit by virtue of our substantial ownership, which will provide opportunities for significant value creation as Rakovina advances its pipeline toward human clinical trials."

As a result of this transaction, Dennis Brown, Ph.D., Edison Oncology’s chairman and Jeffrey Bacha, Edison Oncology’s chief executive officer have been appointed to Rakovina’s Board of Directors. Mr. Bacha has been appointed Rakovina’s executive chairman.

Rakovina was capitalized through a private placement financing and will conduct lead optimization research under a collaboration agreement with the University of British Columbia. Rakovina’s common shares are expected to begin trading as a Tier 2 Issuer on the TSX Venture Exchange under the symbol "RKV" on or about Thursday, April 1, 2021.

Pennsylvania Biotechnology Center (PABC) announces new collaboration and $10 million investment pledge from Daiichi Sankyo, Inc.

On March 31, 2021 The nonprofit Pennsylvania Biotechnology Center (PABC), one of the nation’s most successful life sciences incubators, reported a new collaboration that includes a pledge by Daiichi Sankyo to become an investor in the fund managed by Hatch Biofund Management LLC (Press release, Daiichi Sankyo, MAR 31, 2021, https://www.businesswire.com/news/home/20210331005113/en/Pennsylvania-Biotechnology-Center-PABC-announces-new-collaboration-and-10-million-investment-pledge-from-Daiichi-Sankyo-Inc. [SID1234577465]).

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An investment of $10 million has been committed by Daiichi Sankyo to Hatch Biofund 1, which was created by the Baruch S. Blumberg Institute as a companion to their Incubator and BioAccelerator. The PABC is managed by the Blumberg Institute. Hatch Biofund is currently in a fundraising mode with plans to raise $50 million with a first close within months.

Louis P. Kassa, the PABC’s executive vice president and chief operating officer, said, "Having Daiichi Sankyo join our innovation ecosystem is a big win for the PABC and our nearly 80 member companies, and it’s a win for Bucks County, the Philadelphia region and the Commonwealth of Pennsylvania as well."

Daiichi Sankyo’s interests in oncology and cell and gene therapy align well with the current PABC incubator.

"This collaboration provides Daiichi Sankyo with access to the new therapeutics under development in the incubators as well as a front row seat at the birth of the new cell and gene therapy industry here in our region," Vlad Walko, CEO of Hatch, said. "The lead investment in Hatch reflects Daiichi Sankyo’s confidence in the PABC model and in the Philadelphia region as a major biotech hub."

Daiichi Sankyo, one of the largest pharmaceutical companies in Japan with sales of more than $9 billion, has its U.S. headquarters in Basking Ridge, N.J. "At Daiichi Sankyo, we are committed to exploring and investing in a variety of avenues to improve health and extend lives," said Stu Mackey, Global Head of Business Development, Daiichi Sankyo. "We are proud to partner with the Pennsylvania Biotechnology Center to support the advancement of companies that share our goals for better global human health."

ARTMS Inc. Submits Drug Master File for the First Ever Multi-Curie Production of Gallium-68 with ARTMS’ Solid Target Solution on Medical Cyclotrons

On March 31, 2021 ARTMS Inc. (ARTMS) reported the submission of a Drug Master File (DMF) to the FDA for the high-volume production of Gallium-68 (68Ga) with ARTMS’ solid target technology in combination with a medical cyclotron (Press release, ARTMS Products, MAR 31, 2021, View Source [SID1234577464]). ARTMS’ proprietary QUANTM Irradiation system (QISTM) demonstrated world record production of over 10 Ci 68Ga in 2019 on a low energy cyclotron. These low energy cyclotrons are installed in hundreds of sites globally where the QIS can be installed for daily use.

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"This submission to the FDA demonstrates how the team at ARTMS is executing our strategy. The molecular imaging community has and will continue to develop a number of innovative agents that are labeled with 68Ga. Unfortunately, the current supply chain of germanium/gallium generators is not robust enough to meet projected demand for these products. The action taken today by ARTMS marks a significant step toward solving the forthcoming supply issues for this critical isotope. ARTMS has spent the last 18 months solidifying partnerships with drug developers and building a network of cyclotron operators with the intention of changing the way isotopes are delivered to patients in greatest need of these imaging studies. The ARTMS platform goes far beyond 68Ga production, expanding our client’s reach to other high demand diagnostic and therapeutic isotopes. I am extremely excited about the opportunity that is in front of ARTMS and our partners," says Charles S. Conroy, Chief Executive Officer of ARTMS.

The use of 68Ga labelled products is among the most important innovations in the history of nuclear medicine. Currently, 68Ga radiopharmaceuticals are used to identify and stage a variety of oncology conditions, including neuroendocrine tumors and prostate cancer. This isotope has been traditionally supplied by generators which have limited output capabilities and are logistically and economically burdensome. While generators will continue to be a part of the 68Ga supply chain, ARTMS’ technology will alleviate widespread shortages as demand increases for 68Ga labelled diagnostic agents.

"It is our strategy to work alongside the radiopharmaceutical innovator community to allow the ARTMS solution and associated regulatory filings to be included in the innovator’s investigational new drug and new drug applications," said Dr. Michael Cross, Chief Operating Officer and project lead at ARTMS. "Moving forward, ARTMS will continue to expand collaboration efforts with the radiopharmaceutical innovators to validate ARTMS’ 68Ga with these important medical products."

With a focus on optimizing production potential, ARTMS will continue the development of cyclotron-produced 68Ga and other vitally important medical isotopes, while also pursuing multiple regulatory approvals. ARTMS has a variety of solid targets commercially available used in combination with ARTMS’ QUANTM Irradiation System (QIS).

Gracell Biotechnologies Announces Enrollment of First Patient in Registrational Phase 1/2 Clinical Study for GC007g, an Allogeneic CAR-T Cell Therapy for the Treatment of Relapsed or Refractory B-ALL

On March 31, 2021 Gracell Biotechnologies Inc. (NASDAQ: GRCL) ("Gracell"), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported that they have enrolled the first patient in their pivotal Phase 1/2 clinical study of GC007g, an allogeneic donor-derived anti-CD19 chimeric antigen receptor (CAR)-T cell therapy for the treatment of B-cell acute lymphoblastic leukemia (B-ALL) (Press release, Gracell Biotechnologies, MAR 31, 2021, View Source [SID1234577463]).

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GC007g is an allogeneic HLA (human leukocyte antigen)-matched donor-derived CAR-T therapy. Gracell obtained IND approval for GC007g for the treatment of B-ALL from China’s National Medical Products Administration (NMPA) and the approval for the pivotal Phase 1/2 clinical study in December 2020. The open-label, single-arm Phase 1/2 study is evaluating the safety and efficacy of GC007g in r/r B-ALL patients.

"We are thrilled to announce the enrollment of the first patient into our registrational Phase 1/2 trial for the allogeneic donor-derived CD19-targeted CAR-T therapy, GC007g, for the treatment of patients with B-ALL," said Dr. Martina Sersch, M.D., Chief Medical Officer of Gracell. "GC007g is a unique treatment approach for B-ALL patients who relapse after allogeneic stem cell transplantation and are not eligible for standard-of-care. With Gracell’s innovative portfolio, we are excited to bring novel CAR-T therapies to more patients with high unmet medical need."

About GC007g

GC007g is a donor-derived CD19-directed allogeneic CAR-T cell therapy that has been studied for the treatment of r/r B-ALL in a completed investigator-initiated Phase 1 trial in China, where CAR-T cells were manufactured using T cells from an HLA-matched healthy donor.

About B-ALL

B-ALL, a major form of acute lymphoblastic leukemia (ALL), is one of the most common forms of cancer in children between the ages of two and five and adults over the age of 50.[1] In 2015, ALL affected around 837,000 people globally and resulted in 110,000 deaths worldwide.[2] It is also the most common cause of cancer and death from cancer among children.