February 2022 Update

On February 11, 2022 Ichnos Sciences reported that innovation in medicine by developing potentially transformative biologic treatments in immuno-oncology (Press release, Ichnos Sciences, FEB 11, 2022, View Source [SID1234608006]). The company, currently a subsidiary of Glenmark Holding, SA, plans to pursue external financing following achievement of clinical proof of concept for its lead assets.

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Headquartered in New York City, Ichnos has discovery and manufacturing operations at two sites in Switzerland. As a fully integrated biotechnology company with approximately 225 employees, Ichnos has strong capabilities in research, antibody engineering, CMC and clinical development of biotechnologies.

Ichnos is guided by an accomplished management team with experience developing immune cell engagers within the biopharmaceuticals industry, and is led by Cyril Konto, M.D., President and Chief Executive Officer.

The proprietary BEAT technology platform1 is the basis for Ichnos’ clinical-stage oncology pipeline. Using this technology coupled with the proprietary common light chain library, the company is developing novel multispecific immune cell engagers and modulators, with the goal of realizing its mission to provide breakthrough, potentially curative therapies that may extend and improve lives, writing a new chapter in healthcare.

ONCOLOGY PIPELINE
The first wave of Ichnos’ multispecific antibody pipeline consists of five programs targeting a range of hematologic malignancies and solid tumor indications through engagement of a broad spectrum of immune cells. The most advanced program is ISB 1342, a clinical-stage, potentially first-in-class bispecific antibody targeting CD38 and CD3, which is in Phase 1 for the treatment of relapsed/refractory multiple myeloma. OVERVIEW OF SELECT ONCOLOGY DRUG PRODUCT CANDIDATES ISB 1342 (CD38 X CD3 BISPECIFIC ANTIBODY)

• A Phase 1, open-label, dose-escalation, first-in-human study of ISB 1342 in patients with relapsed/refractory multiple myeloma is ongoing. + Enrollment of patients receiving a weekly dosing regimen is ongoing. + Number of sites participating in the study was expanded in the end of 2021 to enhance enrollment. New locations in the U.S. were added and 11 sites have opened for enrollment in France and are now recruiting subjects. + Clinical proof of concept in the ongoing study is anticipated in the middle of calendar year 2022.
• The primary objectives of the study are to: + Determine maximum tolerated dose and/or recommended Phase 2 dose of ISB 1342 (Part 1 dose escalation). Assess anti-myeloma activity of ISB 1342 according to the International Myeloma Working Group response criteria (Part 2 dose expansion).
• Preclinical data on ISB 1342 were presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting and EHA (Free EHA Whitepaper) 2021 Virtual Congress.
• ISB 1342 was granted Orphan Drug Designation for multiple myeloma by the FDA.
• The bulk drug substance is manufactured at the Ichnos site in La Chaux-de-Fonds, Switzerland. ISB 1442 (CD38 X CD47 BISPECIFIC ANTIBODY)
• This first-in-class 2+1 biparatopic bispecific antibody targeting CD38 x CD47 was generated using the BEAT 2.0 technology developed by scientists in Ichnos’ laboratories in Lausanne at the Biopole life sciences campus.
• ISB 1442 is designed to kill CD38-expressing tumor cells through inhibition of the CD47-SIRPα axis to increase antibody-dependent cellular phagocytosis (ADCP) and enhance antibody-dependent cellular cytotoxicity through complement dependent cytotoxicity (CDC) and antibody-dependent cell cytotoxicity (ADCC), enabled by the architecture and engineered Fc of the molecules.
• IND-enabling studies are proceeding, and IND filing is planned for second quarter of calendar year 2022. A Phase 1/2 first-in-human dose-finding study of ISB 1442 in relapsed/refractory multiple myeloma and other select hematologic malignancies is currently planned to start in the middle of 2022.
• Preclinical data on ISB 1442 were shared in an oral presentation at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting on December 11, 2021. These data, which may be viewed at this link, show: + Higher potency in vitro for ISB 1442 relative to daratumumab in CD38 high/low tumor models as measured by a multiple antibody-dependent mechanisms of action killing assay + Higher tumor growth inhibition for ISB 1442 than daratumumab in CD38 high preclinical in vivo models + Low on-target off-tumor binding with ISB 1442 compared to anti-CD47 mAb (5F9), resulting in lower red blood cell depletion and potentially a better therapeutic index than anti-CD47 bivalent monoclonal antibodies
• The first bulk drug substance batches to support IND filing and early clinical studies were manufactured at the Ichnos site in La Chaux-de-Fonds, Switzerland in 2021.Based on BEAT 2.0 technology, ISB 2001 trispecific antibody (TREATTM) represents a first-in-class potential treatment for hematologic malignancies and is designed to extend therapeutic durability.
• Identification and amino acid sequence lock of the top two candidates was achieved in 2021. Preclinical evaluation of in vivo efficacy, PK/PD correlation, additional biophysical properties description, late pharmacology studies and other attribute-defining studies are ongoing this quarter, and the results will inform the selection of the drug product candidate in the first half of calendar year 2022.
• Process development is ongoing at the Ichnos site in La Chaux-de-Fonds, Switzerland. AUTOIMMUNE DISEASES Ichnos has two monoclonal antibody drug product candidates addressing autoimmune diseases in the pipeline. The first, ISB 880, an anti-IL-1RAP antagonist, was licensed to Almirall, S.A. in December 2021, and the second, ISB 830 (telazorlimab), an OX40 antagonist that completed a Phase 2b study in moderate to severe atopic dermatitis in calendar year 2021, is in out-licensing discussions.

Both compounds have potential across a range of autoimmune diseases and are being out-licensed to enable a greater focus on oncology. Ichnos entered an exclusive global licensing agreement for ISB 880 in autoimmune diseases with Almirall in December 2021. Within the terms of the agreement, Almirall will assume full cost and responsibility for the global development and commercialization of the compound. Ichnos received an upfront payment of €20.8 million and the deal also includes development and commercial milestone payments and tiered royalties based upon future global sales.

• ISB 880, a fully human, high-affinity, monoclonal antibody blocking IL-1RAP signaling, has completed IND-enabling studies for patients with autoimmune diseases. The optimal antibody profile, the strong in vitro and in vivo data package, as well as toxicology, CMC, and clinical pharmacology plans will enable U.S. IND filing by Almirall in the first half of calendar year 2022.
• Blockade of IL-1RAP simultaneously abrogates multiple disease drivers among the IL-1 family of proinflammatory cytokine receptors, including IL-1R, IL-33R, and IL-36R, differentiating ISB 880 from single cytokine blockade therapies. These cytokines have been implicated in numerous autoimmune conditions, opening opportunities for ISB 880 to be positioned across broad disease indications.
• To date there is no IL-1RAP antagonist approved or under clinical development for autoimmune disease, positioning ISB 880 as a potential first-in-class therapeutic.
• Ichnos will retain rights for antibodies acting on the IL-1RAP pathway for oncology indications.The database for the ISB 830-204 Phase 2b clinical study in atopic dermatitis was locked in October 2021. This study, which was conducted in the U.S., Canada, Germany, Czech Republic, and Poland, had a randomized, controlled, multicenter design and assessed three doses and two dosing schedules of telazorlimab versus placebo in adults with moderate-to-severe atopic dermatitis (AD).
• Results from the double-blind portion of the study are summarized below. + Efficacy: The primary endpoint of EASI score, % change from baseline to Week 16, was achieved for the two highest doses of telazorlimab tested (300 mg and 600 mg q 2 weeks) versus placebo. Numerical improvements were also seen for the two higher dose arms of telazorlimab compared to placebo in the secondary endpoints of EASI75 and Investigator Global Assessment, but most of the differences were not statistically significantSafety: Telazorlimab was well tolerated.

The most commonly reported adverse events (>5%) were atopic dermatitis, nasopharyngitis, upper respiratory tract infection, and headache. One patient with pre-existing hypertension in the telazorlimab group died due to a presumed cardiovascular event during the treatment period. The investigator considered the death to be unrelated to the study drug.
• In addition to data from the 16-week primary analysis period, preliminary results from the open-label extension and follow-up period of this study, which was ongoing at the time, were presented at the 2021 Society for Investigative Dermatology Virtual Meeting and are accessible here. Of note: Clinical efficacy continued to improve after Week 16, with maximal impact achieved several weeks later + Reduction in AD disease activity was maintained after discontinuation of telazorlimab, through three months of follow-up
• A U.S. IND to conduct studies of telazorlimab in autoimmune diseases, including Rheumatoid Arthritis (RA), is active. • Licensing discussions are ongoing.

NeuBase Therapeutics Reports Business Update and Financial Results for the First Quarter of Fiscal Year 2022

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.

LinKinVax raises €4.350 Million in Funding to accelerate the development of its innovative vaccine platform

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).

Ucello Therapeutics Completes US$25 Million Series A Financing

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."

Apmonia Therapeutics announces a 4 M€ fundraising round

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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