On February 11, 2022 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported its financial results for the three-month period ended December 31, 2021, and other recent developments (Press release, NeuBase Therapeutics, FEB 11, 2022, View Source [SID1234607987]).

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"We are making significant progress in advancing the IND-enabling studies for the development candidate for our myotonic dystrophy type 1 (DM1) program, and we expect to file an IND with the FDA in Q4 CY2022," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. "These studies are on track for data readouts to occur throughout CY2022, with the first presentation of rodent pharmacokinetic and bioavailability data to occur at an upcoming scientific meeting. We expect these data to illustrate the differentiated potential for our candidate to be a whole-body solution to treat DM1 and the unique ability of our delivery shuttle for distribution into the brain. The ability to cross the blood brain barrier and reach the deep brain is also especially relevant for our Huntington’s disease program, where we are planning to initiate scale-up and toxicology activities this year."

Dr. Stephan added, "In addition, I’m especially excited to have welcomed Todd to the executive team as Chief Financial Officer. The team and science are strong, and I believe we are at a pivotal moment for NeuBase as we are building a robust data package that is expected to support bringing our first candidate into the clinic for DM1, validate our genetic medicine technology platform to efficiently deliver genetic medicines with broad tissue distribution, including into the deep brain, and to precisely engage genetic mutations in a manner that is well-tolerated with the potential for sustained efficacy."

First Quarter of Fiscal Year 2022 and Recent Operating Highlights

Myotonic Dystrophy Type 1 (DM1) Program: NeuBase is making steady progress advancing IND-enabling studies for its development candidate in the DM1 program, which includes PK, absorption, distribution, metabolism, and excretion (ADME), and bioavailability via intravenous (IV) and subcutaneous (SQ) routes of administration, exploratory and IND-enabling Good Laboratory Practice (GLP) toxicology, and mechanism of action studies. In addition, GMP manufacturing of NeuBase’s development candidate to support Phase 1/2 clinical trials has been successfully implemented via contract manufacturing organizations. The Company plans to announce a robust data package through posters and presentations at scientific meetings and peer-reviewed publications throughout CY2022. NeuBase expects these data to support the submission of an IND filing to the FDA in the Q4 CY2022.
Huntington’s Disease (HD) Program: The HD program is currently in preclinical development. In CY2022, NeuBase expects to nominate a development candidate and initiate scale-up and toxicology activities to support an IND filing to the FDA in CY2023.
KRAS Oncology Program: The Company is conducting in vitro mechanistic studies and in vivo pharmacology studies for the KRAS program (KRAS G12V and G12D mutations).
Appointed New Chief Financial Officer: The Company appointed Todd P. Branning as CFO. Mr. Branning brings more than 25 years of experience leading corporate finance and accounting, tax, financial planning and analysis, and investor relations for several publicly traded pharmaceutical companies.
Financial Results for the Fiscal Quarter Ended December 31, 2021

As of December 31, 2021, the Company had cash and cash equivalents of approximately $47.3 million, compared with approximately $52.9 million as of September 30, 2021.
NeuBase estimates its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures into the first quarter of CY2023.
For the fiscal quarter ended December 31, 2021, the Company reported a net loss of approximately $7.7 million, or a net loss of $0.24 per share, compared with a net loss of approximately $4.1 million, or a net loss of $0.18 per share, for the same period last year.
For the fiscal quarter ended December 31, 2021, total operating expenses were approximately $7.3 million, consisting of approximately $2.9 million in general and administrative expenses and $4.4 million of research and development expenses. This compares with total operating expenses of approximately $4.7 million for the same period last year, consisting of approximately $2.7 million in general and administrative expenses and $2.0 million in research and development expenses.

On February 10, 2022 LinKinVax, a clinical-stage biotechnology company, reported completion of a €4.350 million seed capital funding round (Press release, LinKinVax, FEB 10, 2022, View Source [SID1234648589]). It was led by LinKinVax’s core shareholders and business angels and will enable LinKinVax to accelerate the clinical development of its innovative protein-based vaccine platform, which can be tweaked to changes and mutations in target pathogens.
LinKinVax has already risen to prominence in the French sector, with Bpifrance granting it €31 million under a Covid-related PIA PSCP program and Government "Plan Relance", in May 2021.
LinKinVax’s innovative technology directly targets dendritic cells ("DC"), which play a crucial role in the immune system, stimulating and regulating immune responses. It is predicated on work by the U955 research team at the Vaccine Research Institute/Inserm (VRI) in which Inserm, University of Paris Est Créteil (UPEC), and the Mondor Institute of Biomedical Research (IMRB) participate. Thanks to the potential of its technology platform and the advances in its portfolio, LinKinVax aims to make a decisive contribution to the global public health challenges of infectious diseases and cancer.

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André-Jacques Auberton-Hervé, Chairman co-founder and CEO of LinKinVax commented:

"We are thrilled that our breakthrough vaccine technology has generated so much interest among new investors, and with the renewed trust our longstanding partners, we can push ahead confidently with the next stages of our ambitious clinical program. Our objective is to provide effective vaccines as rapidly as possible to help address the public health challenges linked to the various pathogens we are targeting—HIV, SarsCov2, HPV, papillomavirus-related cancers, chlamydia."

The investors who contributed to the round are: André-Jacques Auberton-Hervé (co-founder and CEO of LinKinVax), Jean-Paul Kress (CEO of Morphosys), Geoffrey Duyk (US biotechnology entrepreneur and investor), Giorgio Anania, Rémi Gaston-Dreyfus and Alain Tingaud (recognized entrepreneurs in the high-tech sector).

On February 10, 2022 Ucello Therapeutics ("Ucello"), a biotechnology company developing universal CAR-T therapies, reported the successful completion of a US$25 million Series A financing (Press release, Ucello Therapeutics, FEB 10, 2022, View Source [SID1234609738]).

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The funding round was led by Matrix Partners China, along with other investors Co-Win Ventures, Northern Light Venture Capital, and Elikon Venture. Also joining the round was existing angle investor Chengdu Bio-town Fund.

Proceeds from the financing will be used to advance the company’s development of proprietary universal CAR-T technology platform CBT-X20, progress pipeline programs including lead asset AT19 through to US and China IND filings, and build a state of the art GMP-ready clinical manufacturing facilities.

"We are delighted to have achieved this significant milestone, which allows us to welcome several top caliber investors such as Matrix Partners China, Co-Win Ventures, Northern Light Venture Capital, Elikon Venture, and Chengdu Bio-town Fund," said Dr. Qiang Zou, Chairman and CEO of Ucello. "With this funding, we will further accelerate our goal of developing breakthrough universal CAR-T therapies and move our pipeline towards clinical development where we hope to help patients worldwide as soon as possible."

"Cell therapy is a fast-moving field and the next generation of therapies could be off-the-shelf in nature," said Matrix Partners China partner Dr. Eric Yu. "Allogeneic cell therapy, like the universal CAR-T that Ucello is developing, could potentially address huge unmet medical needs by making cell therapy a lot more accessible and readily available. Ucello’s universal CAR-T cell therapy platform has demonstrated great potential through excellent early clinical results in investigator-initiated trials with global leading safety and efficacy profiles. We believe the company is well-positioned to bring benefits to patients in China and globally as early as possible."

"Ucello’s products at this stage have demonstrated unparalleled advantages for patients who have previously received bone marrow transplants, as well as for those whose conditions continue to progress after autologous CAR-T treatment. It is not only cost-controlled, but also truly meets the clinical need for timeliness," said Dr. Xin Huang, Managing Partner of the Co-Win Ventures. "This obvious advantage in performance is mainly thanks to the team’s commitment to benefits for patients alongside years of research and deep knowledge in the field of cord blood-derived CAR-T cell technology. We are pleased to have the opportunity to help Ucello build on its success and accelerate the clinical application of its products to bring sustainable clinical benefits to more patients."

"Universal CAR-T is a very competitive field for players in the cell therapy sector, in which few breakthroughs having been made so far due to many technical hurdles," stated Mr. Feng Deng, Founding and Managing Partner of the Northern Light Venture Capital. "With a deep understanding of the immunotherapy and years of continuous efforts, the Ucello team has developed a unique platform technology which started to produce excellent clinical outcome with great potential to develop future blockbusters. We are very pleased to continually support the development of Ucello."

Dr. Jianfeng Zhou, Director of the Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, said

"Universal CAR-T cell therapy is the current focus and hotspot of clinical research in the field of hematological oncology worldwide. We expect Ucello to achieve breakthroughs in this highly challenging field in both China and globally."

On February 10, 2022 Apmonia Therapeutics reported a 4 M€ fundraising round to complete regulatory preclinical studies of its anti-cancer immunotherapy drug candidate (AP-01) Success for Apmonia Therapeutics’ technology platform developing a portfolio of therapeutic peptides targeting tumor microenvironment in solid tumors (Press release, Apmonia Therapeutics, FEB 10, 2022, View Source [SID1234609536]).

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On February 10, 2022, Twist Bioscience Corporation (the "Company") reported that entered into an underwriting agreement (the "Underwriting Agreement") with J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC, and Cowen and Company LLC, as the representatives of the several underwriters named therein (collectively, the "Underwriters"), pursuant to which the Company agreed to issue and sell an aggregate of 5,227,272 shares of its common stock, including 681,818 shares pursuant to an option granted to the Underwriters to purchase such additional shares that was exercised in full (collectively, the "Shares"), to the Underwriters (the "Offering") (Filing, 8-K, Twist Bioscience, FEB 10, 2022, View Source [SID1234608109]). The Shares will be sold to the Underwriters at a public offering price of $55.00 per share, less the underwriting discount and commissions. The Underwriting Agreement contains customary representations and warranties, conditions to closing, market standoff provisions, termination provisions and indemnification obligations, including for liabilities under the Securities Act of 1933, as amended.

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The Offering is being made pursuant to the Company’s registration statement on Form S-3 (Registration No. 333-238906), which was filed with the Securities and Exchange Commission (the "SEC") on June 3, 2020, and the prospectus supplement dated February 10, 2022 filed by the Company with the SEC. The Offering is scheduled to close on or about February 15, 2022, subject to customary closing conditions.