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Exscientia Completes $100 million Series C Financing Round With BlackRock Funds

On March 4, 2021 Exscientia, a clinical stage pharmatech pioneering the use of artificial intelligence (AI) to design new drugs, reported that funds managed by BlackRock joined the Company’s Series C investment round (Press release, Exscientia, MAR 4, 2021, View Source [SID1234576073]). Including existing Series C investors, Novo Holdings, Evotec, Bristol Myers Squibb, and GT Healthcare Capital, the round totalled $100 million in funding.

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This new capital will be used to support Exscientia’s platform development towards autonomous drug design. In addition, the company will extend its proprietary pipeline into clinical trials and expand existing capabilities in biological analytics that support target selection and portfolio development.

With the company’s end-to-end AI-first drug discovery platform, CentaurAI, Exscientia has been able to generate novel drugs and overcome conventional drug discovery limitations several years faster than industry benchmarks. Exscientia has demonstrated the platform’s capabilities by creating the first fully AI-designed drug to enter clinical trials and advancing multiple drug candidates into preclinical testing. In addition to its growing proprietary pipeline, the company has conducted drug discovery partnerships with Bristol-Myers Squibb, Sanofi, Bayer and Dainippon Sumitomo as well as several biotech companies.

"Exscientia is breaking ground in small molecule drug design, with a platform that radically improves drug discovery" says William Abecassis, Head of BlackRock’s Innovation Capital who will join Exscientia’s Board of Directors as an observer. "We are thrilled to be investing in this world-class team, who are already delivering results with AI-designed drugs now entering clinical trials."

Andrew Hopkins, CEO of Exscientia added, "We are delighted that BlackRock shares our vision for revolutionising how drugs are discovered. It is also recognition of the ingenuity and hard work of our employees to turn the promise of AI into reality today, where we are bringing the world’s first AI designed drugs into the clinic. BlackRock’s investment is an important step in our vision that all drugs will be designed by AI. I believe that our company’s reimagined approach to drug discovery will become the new de facto standard."

Exscientia has doubled in size over the past year and expects to double again in 2021, including a recent US expansion into Miami and Boston. The company employs over 100 people from over 20 different nations, with equal balance of technologists and drug discovery scientists, of which 60% are PhDs. Exscientia is named the fastest growing private biotech or pharmaceutical company in the UK in the 2021 Alantra Pharma Fast 50, as well as one of the fastest growing healthcare companies in the Financial Times FT1000.

X4 Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Corporate Update

On March 4, 2021 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, X4 Pharmaceuticals, MAR 4, 2021, View Source [SID1234576072]). The company also provided an update on its lead product candidate, mavorixafor, a novel small molecule currently being evaluated in a Phase 3 clinical trial (4WHIM) for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials for patients with Waldenström’s macroglobulinemia (Waldenström’s) and Severe Congenital Neutropenia (SCN), respectively.

"As many companies did, we encountered a number of unexpected challenges in 2020, most notably the COVID-19 pandemic. However, I could not be prouder of our accomplishments, as we remained steadfast in our commitment to advance our lead candidate, mavorixafor, in a number of rare disease indications," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "We believe that we have much to look forward to this year, as we have overcome these obstacles, have made significant progress in advancing our clinical and research programs, and assembled an exceptional team to execute on our 2021 goals and beyond.

"On the clinical front, we continue to expect top-line data from our Phase 3 WHIM trial in 2022 and, as our Phase 1b Waldenström’s trial is progressing well, we continue to expect initial data from that study in the first half of 2021, with a more fulsome dataset expected towards the end of 2021. We also anticipate initial data from our SCN Phase 1b trial in 2021."

Recent Highlights

Received Fast Track and Rare Pediatric Designations (RPD) from the FDA for Mavorixafor for the treatment of WHIM Syndrome: These FDA designations further recognize WHIM as a serious condition with a clear unmet need in both adult and pediatric populations.
Through the Fast Track program, X4 will be eligible for more frequent meetings with the FDA to discuss the drug’s development plan, protocols and clinical data that would support mavorixafor’s potential approval for WHIM.
Under the RPD program, a sponsor who receives an approval for a drug for a "rare pediatric disease" and a Fast Track designation may qualify for a voucher that can be redeemed to receive a priority review by the FDA for any subsequent marketing application for a different product. Such a voucher is transferable and may be sold.
Mavorixafor was previously granted Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by the FDA and the European Commission for the treatment of WHIM syndrome.
Strengthened Leadership Team: During the Fourth Quarter of 2020:
Diego Cadavid, M.D., joined as Chief Medical Officer, bringing more than 20 years of industry experience and having led multiple programs through all phases of clinical development, including small molecules and biologics for the treatment of rare and immunological diseases.
Art Taveras, Ph.D., joined as Chief Scientific Officer with more than 30 years of experience leading small molecule research and development programs focused on cancer, dysregulated immune disorders, neurodegeneration, and metabolic diseases.
Alison Lawton was appointed to the company’s Board of Directors, bringing more than 30 years of experience across a full spectrum of drug development and commercial roles and having previously served on X4’s corporate advisory board and as consulting Chief Operating Officer.
Fourth Quarter 2020 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $80.7 million in cash, cash equivalents and restricted cash as of December 31, 2020. X4 continues to expect that its cash and cash equivalents will fund company operations into 2022.
Research and Development Expenses were $12.3 million and $41.9 million for the fourth quarter and full year ended December 31, 2020, respectively, as compared to $7.1 million and $30.2 million for the comparable periods in 2019, respectively. R&D expenses include $0.6 million and $2.3 million of certain non-cash expenses for the fourth quarter and fully year ended December 31, 2020, respectively.
General and Administrative Expenses were $5.4 million and $20.9 million for the fourth quarter and full year ended December 31, 2020, respectively, as compared to $3.9 million and $17.6 million for the comparable periods in 2019, respectively. G&A expenses include $0.8 million and $3.1 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2020, respectively.
Net Loss: X4 reported net losses of $18.4 million and $62.1 million for the fourth quarter and full year ended December 31, 2020 as compared to net losses of $10.8 million and $52.8 million for the comparable periods in 2019, respectively. Net losses include $1.4 million and $5.4 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2020, respectively. Net losses in the full year of 2019 included a $3.9 million loss on the sale of non-financial assets.
Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 1053189. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

PORTAGE BIOTECH DIVESTS PORTAGE PHARMACEUTICALS LIMITED TO JUVENESCENCE LTD

On March 4, 2021 Portage Biotech Inc. (NASDAQ: PRTG) (CSE: PBT.U) ("Portage" or the "Company") a clinical stage immuno-oncology company accelerating research and development to overcome immune resistance, reported the divestment of its three legacy businesses; Portage Pharmaceuticals Limited (PPL), including subsidiaries Portage Glasgow Ltd. and EyGen Ltd, to Juvenescence Ltd., a company developing therapies to transform the way people age (Press release, Portage Biotech, MAR 4, 2021, View Source [SID1234576071]). This asset disposition reflects a continuum of Portage’s business strategy to focus on its pipeline of immuno-oncology platforms.

In exchange for the assets, the out-licensing agreement states that Portage may be entitled to receive up to USD $244 million in future milestone payments based on specified development criteria. The Company will also be eligible to receive royalties in the future on global net sales of products developed utilizing the PPL intellectual property.

Dr. Ian Walters, chief executive officer of Portage, commented, "We are dedicated to the continued development of our immuno-oncology pipeline and the progression of our three products in clinical testing. Today’s transaction reflects this commitment and enables us to direct both our personnel and capital resources towards our immuno-oncology programs while maintaining upside in the event that the assets developed using PPL’s intellectual property are successfully commercialized."

This transaction is considered a related party transaction within the meaning of Multilateral Instrument 61-101 Protection of Minority Security Holders in Special Transactions ("MI 61-101"). The Company is relying on appropriate exemptions from the formal valuation and minority shareholder approval requirements of MI 61-101 in respect of these issuances.

INOVIO to Present at Upcoming Investor Conferences in March

On March 4, 2021 INOVIO (NASDAQ:INO), a biotechnology company focused on bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases and cancer, reported that Dr. J. Joseph Kim, President and CEO, along with other members of INOVIO management, will present at the following investor conferences in March (Press release, Inovio, MAR 4, 2021, View Source [SID1234576070]):

H.C. Wainwright Global Life Sciences Conference
Date: Tuesday, March 9, 2021
Virtual 1×1 meetings only

Oppenheimer 31st Annual Healthcare Conference
Date: Thursday, March 18, 2021
Time: 10:40 a.m. ET
Presentation Format: Corporate Presentation

Live and archived versions of the virtual presentations will be available through the INOVIO Investor Relations Events page and may be accessed by visiting INOVIO’s website at View Source All presentation times are subject to change.

Replimune to Present at Three Upcoming Investor Conferences

On March 4, 2021 Replimune Group, Inc. (Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported that members from the Replimune management team will present and host one-on-one meetings at the following three virtual investor conferences (Press release, Replimune, MAR 4, 2021, View Source [SID1234576069]).

H.C. Wainwright Global Life Sciences Conference
Date: March 9-10, 2021
Pre-Recorded Formal Presentation will be made live: Tuesday, March 9, 2021 at 7:00 am ET

Barclays Global Healthcare Conference
Date: Thursday, March 11, 2021
Fireside Chat Presentation Time: 9:10 am ET

33rd Annual Roth Conference
Date: Wednesday, March 17, 2021
Presentation Time: 1:30 pm ET

A simultaneous webcast of the fireside chat presentation at the Barclays Global Healthcare Conference will be available in the Investors section of Replimune’s website at www.replimune.com. A replay will be available for approximately 90 days following the conference.