On December 23, 2021 Oncorena, developing a potential breakthrough therapy for advanced renal cancer, reported that it receives a capital injection of MSEK 66 from one of the company’s principal shareholders together with two new investors (Press release, Oncorena, DEC 23, 2021, View Source [SID1234597684]). Under the terms of the agreement, Oncorena can receive an additional SEK 94 million in the future. The capital will primarily fund Oncorena’s first clinical study, a phase I/II study with orellanine in patients with advanced kidney cancer undergoing dialysis.

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The current shareholder HealthCap has together with the two new investors, Linc AB and Fåhraeus Startup and Growth AB, invested a total of MSEK 66 in new capital to finance the first part of the Oncorena’s Phase I/II study. If the first part of the Phase I/II study shows positive results (Proof of Concept), the parties intend to invest an additional sum of MSEK 94 in the second part of the study.

HealthCap, one of the largest life science venture capital funds in Europe, is since 2016 one of Oncorena’s largest shareholders. The investment company Linc AB is listed on Nasdaq Stockholm and invests in product oriented Nordic life science companies, primarily in pharma and medtech companies. Fåhraeus Startup and Growth AB is a newly founded venture capital fund focusing on early investments in life science and tech companies. The investment is subject to approval by an Extraordinary General Meeting to be held in January 2022.

Earlier this year, the Swedish Medical Products Agency approved Oncorena’s first clinical trial of orellanine in patients with advanced renal cancer undergoing dialysis. Orellanine is a substance with a unique mode of action that has demonstrated specific and powerful anti-tumour effects on advanced renal cancer in a number of preclinical models.

"We are grateful for this capital injection that enables us to get necessary and crucial results that will be decisive for Oncorena’s continued clinical development of orellanine and new ventures in the field of kidney cancer. We also hope that the results from the upcoming clinical study will be of great benefit to patients in the future," said Lars Grundemar M.D., Ph.D., Chief Executive Officer of Oncorena.

"It is gratifying to announce that Oncorena is now entering a new stage with a capital injection of up to a total of MSEK 160 in a Serie A-round from three strong life science investors. With the financing in place, Oncorena can now focus on exploiting the potential of the company’s innovation in kidney cancer, developing the company further and accelerating the growth journey," said Andreas Segerros, Oncorena’s Chairman of the Board.

About the Phase I/II clinical trial
The Phase I/II clinical trial of orellanine will enrol patients with advanced renal cancer already on dialysis due to renal failure. The study will be conducted at the Centre for Clinical Cancer Studies at the Karolinska University Hospital in Stockholm, Sweden, and will study safety, tolerability, pharmacokinetics and signs of anti-tumour effects in treatment with a synthetic form of orellanine. The Phase I/II trial will include up to 40 patients and may include patients from other European countries.

About orellanine
Orellanine, which has a new and unique mode of action, is being developed for organ-specific chemotherapy with curative potential for patients with advanced renal cancer undergoing dialysis. Orellanine is found in mushrooms of the Cortinarius family, these are sometimes accidentally picked and eaten as they are mistaken for funnel chanterelles. The clinical effects of orellanine are well documented and are completely limited to the kidneys.

About kidney cancer
Approximately 400,000 patients are affected by kidney cancer globally according to the WHO. The disease can often be cured by surgery if detected in time, but unfortunately the diagnosis is often made when the tumour has already spread to other organs. The prognosis is then considerably less favourable and certain groups have a median survival of less than two years. Today the disease is treated with various types of targeted and immuno-active drugs, often with severe side effects, and standard chemotherapy drugs have limited effect. There is therefore a great and urgent unmet medical need for new, effective and safe drugs.

On December 23, 2021 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 fiscal year ended September 30, 2021, and other recent developments (Press release, NeuBase Therapeutics, DEC 23, 2021, View Source [SID1234597659]).

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"NeuBase is focused on significantly reducing the burden of untreatable morbidity and mortality caused by rare and common diseases across the globe. To achieve this goal, we designed, built, and validated a new precision genetic medicines platform technology that can uniquely drug the double-stranded human genome and address disease at the root of causality without many of the limitations of early precision genetic medicine technologies. We are poised to file our first Investigational New Drug (‘IND’) applications with the U.S. Food and Drug Administration (‘FDA’) beginning in calendar year 2022 and intend to scale into additional indications with increasing speed and efficiency thereafter," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase.

"This past year, we validated the ability of our technology in proof-of-concept studies to directly drug the double-helix of the human genome, including difficult double-stranded structures of RNA targets, and engage with mutant genes to resolve most causal mechanisms of disease. The validation of our platform’s capabilities included data describing that we have overcome many limitations of early precision genetic medicine technologies, such as biodistribution, tolerability, selectivity, manufacturability, durability, and scalability. We also presented data that our delivery shuttle enables compounds to elicit pharmacologic effects in multiple tissues, including in the brain and muscle, after subcutaneous administration in preclinical animal models," said William Mann, Ph.D., M.B.A., Chief Operating Officer of NeuBase.

"We recently nominated the development candidate for our myotonic dystrophy type 1 (DM1) program, which we believe has the potential to be a best-in-class therapy that offers a patient-friendly route of administration, a whole-body solution for the muscle, heart, and brain manifestations of the disease. Furthermore, the mechanism of action of our development candidate is designed to engage with the toxic RNA hairpin structure to release the splicing proteins, restoring normal RNA splicing and downstream protein production, including DMPK. We have initiated IND-enabling studies for this candidate, with data read-outs expected across CY2022. We expect these data will support the submission of an IND filing to the FDA in the fourth quarter of CY2022," stated Sandra Rojas-Caro, M.D., Chief Medical Officer of NeuBase.

"As a result of the nomination of our DM1 program candidate, we established CMC expertise at our new facility in Cambridge, Massachusetts that is co-located with our clinical development team, finalized the formulation of our development candidate to enable systemic routes, and completed process development. We also scaled-up manufacturing in-house and with contract manufacturing partners to support non-clinical toxicology, product stability, and Phase 1/2 clinical trials," said Tony Rossomando, Ph.D., Chief Technology Officer of NeuBase.

Dr. Stephan concluded, "In parallel, we are making significant progress in our therapeutic program for Huntington’s disease. For example, we have illustrated with preclinical in vivo data that our proprietary delivery technology allows our genome-targeting compounds to advance beyond intrathecal delivery and enabling a systemically administered allele-selective therapy, overcoming challenges seen with other programs. Furthermore, preclinical data show that our PATrOL-enabled compounds can silence activating KRAS point mutations in vivo to inhibit protein production, which has the potential to target G12D and G12V, the two most common and historically ‘undruggable’ cancer-driving point mutations that represent the majority of KRAS tumors. We believe these data set the stage for a potentially first-in-class precision genetic medicine approach for oncology capable of selectively targeting mutations at the single-base level."

Fourth Quarter of Fiscal Year 2021 and Recent Operating Highlights

Myotonic Dystrophy Type 1 (DM1) Program: NeuBase recently nominated its development candidate for the DM1 program and initiated chemistry manufacturing controls ("CMC") scale-up for IND-enabling toxicology and Phase 1/2 clinical trials. In CY2022, NeuBase plans to conduct pharmacokinetic and absorption, distribution, metabolism, excretion (PK/ADME) and bioavailability (IV/SQ), exploratory toxicology, IND-enabling GLP toxicology, and mechanism of action studies, which are expected to support an IND filing to the FDA in the fourth quarter of CY2022.
Huntington’s Disease (HD) Program: The NT-0100 program is currently in preclinical development as a potential treatment for HD. In CY2022, NeuBase expects to initiate scale-up and toxicology activities to support an IND filing to the FDA in CY2023.
KRAS Oncology Program: NeuBase expanded its pipeline into oncology with the advancement of the KRAS program (KRAS G12V and G12D mutations) from concept into in vivo proof-of-principle.
Genetic Target Prioritization: The Company finalized a rank-ordered mutational database. All available monogenic and cancer-causing mutations have been ranked for internal pipeline expansion and prioritize partnering opportunities.
Financial Results for the Fiscal Year Ended September 30, 2021

As of September 30, 2021, the Company had cash and cash equivalents of approximately $52.9 million, compared with approximately $32.0 million as of September 30, 2020
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 year ended September 30, 2021, the Company reported a net loss of approximately $25.4 million, or a net loss of $0.93 per share, compared with a net loss of approximately $17.4 million, or a net loss of $0.89 per share, for the same period last year
For the fiscal year ended September 30, 2021, total operating expenses were approximately $26.6 million, consisting of approximately $12.2 million in general and administrative expenses, $11.5 million of research and development expenses, and $2.9 million in research and development expenses related to the acquisition of assets of Vera Therapeutics, Inc. This compares with total operating expenses of approximately $17.1 million for the same period last year, consisting of approximately $10.1 million in general and administrative expenses and $6.9 million in research and development expenses

On December 22, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that the European Commission has granted Orphan Drug Designation to SNDX-5613, the Company’s highly selective oral menin inhibitor, for the treatment of acute myeloid leukemia (AML) (Press release, Syndax, DEC 22, 2021, View Source [SID1234597607]).

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"Supported by a growing body of clinical data, we firmly believe that SNDX-5613 is ideally positioned to serve as a best-in-class, meaningful intervention for patients with NPM1 and MLLr acute leukemias," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Receipt of Orphan Drug Designation from the European Commission further validates the important role that SNDX-5613 could play in the treatment of this highly underserved patient population, and we are fully committed to ensuring that it is able to reach as many of these patients as possible."

The European Commission grants Orphan Drug Designation for medicinal products intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union (EU) and when no satisfactory method of diagnosis, prevention, or treatment of the condition can be authorized. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market.

SNDX-5613 was previously granted Orphan Drug Designation for the treatment of adult and pediatric AML by the U.S. Food and Drug Administration.

About SNDX-5613

SNDX-5613 is a potent, selective, small molecule inhibitor of the menin-MLL binding interaction that is being developed for the treatment of mixed lineage leukemia rearranged (MLLr) acute leukemias including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), and NPM1 mutant AML. In preclinical models of MLLr acute leukemias, SNDX-5613 demonstrated robust, dose-dependent inhibition of tumor growth, resulting in a marked survival benefit. Menin-MLL interaction inhibitors have also demonstrated robust treatment benefit in multiple preclinical models of NPM1 mutant AML, which represents the most frequent genetic abnormality in adult AML. SNDX-5613 is currently being evaluated in the Company’s AUGMENT-101 Phase 1/2 open-label clinical trial for the treatment of relapsed/refractory (R/R) acute leukemias. SNDX-5613 was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Commission for the treatment of patients with AML, and Fast Track designation by the U.S. FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a mixed lineage leukemia rearranged MLLr or NPM1 mutation.

On December 22, 2021 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688), a patient-focused, innovative, commercial-stage, global biopharmaceutical company, reported the promotion of Harald Reinhart, M.D., from his current role as Chief Medical Officer (CMO) to President and Head of Global Development for Neuroscience, Autoimmune and Infectious Diseases (Press release, Zai Laboratory, DEC 22, 2021, View Source [SID1234597609]). Dr. Reinhart has been with the company since inception, first as an advisor and since 2017, as Chief Medical Officer responsible for the autoimmune and infectious diseases portfolio.

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"Harald has played a pivotal role in building Zai Lab’s broad and deep pipeline in autoimmune, infectious diseases and neuroscience," said Dr. Samantha Du, Founder, Chairperson and CEO at Zai Lab. "This promotion not only recognizes Harald’s tremendous contributions but will also empower him to further build a leading clinical team across these therapeutic areas."

Prior to joining Zai Lab, Dr. Reinhart served at Shionogi U.S. as Senior Vice President and Head of Clinical Development and Medical Affairs directing a broad portfolio of antibiotics, diabetes, allergy, and pain medications. Between 2003 and 2011, he was with Novartis as Vice President and Therapeutic Area Head, Clinical Development and Medical Affairs, where he oversaw successful regulatory filings for Coartem, Famvir, Sebivo, and Cubicin, and managed clinical development teams for infectious disease, immunity, transplantation, and renal disease. At Bayer Corp, he was international clinical project lead for ciprofloxacin and acarbose, and managed various other compounds.

Dr. Reinhart holds a doctorate in medicine from the University of Würzburg in Germany where he trained in surgery, anesthesiology, and internal medicine. He completed his medical and subspecialty training in the U.S. with board-certifications in internal medicine and infectious diseases. He is currently on faculty at Yale School of Medicine as Adjunct Clinical Professor of infectious diseases.

"It has been a phenomenal experience working at Zai Lab for the last seven years helping to bring transformative medicines to patients. I’m grateful for Samantha’s support and look forward to developing more medicines that truly make a difference in the lives of patients," said Dr. Reinhart. "I feel privileged to work every day with world-class colleagues at Zai Lab who share a unified vision for innovative drug development."

On December 22, 2021 Sandoz, a global leader in generic and biosimilar medicines, reported that it has submitted a Marketing Authorization Application for a proposed biosimilar trastuzumab (150 mg, for intravenous use) developed by EirGenix, Inc. to the European Medicines Agency (EMA) (Press release, Sandoz, DEC 22, 2021, View Source [SID1234597614]).

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Trastuzumab is a monoclonal antibody used for the treatment of human epidermal growth factor receptor 2 positive (HER2-positive) breast cancer and metastatic gastric cancers2. Sandoz is seeking approval for the same indications as the reference medicine, based on a comprehensive package that includes analytical, preclinical, and clinical data.

"In 2020, breast cancer accounted for 28.7% of all new cancer cases diagnosed, making it the most frequently occurring cancer and first cause of cancer death among women in Europe1", said Florian Bieber, Global Head of Biopharmaceuticals Development, Sandoz. "Biosimilars have enormous potential to improve cancer care. Today’s submission is an encouraging step forward in our mission to expand access to advanced biologics treatments to address the evolving needs of patients, healthcare professionals and healthcare systems."

As part of the license agreement signed in April 2019, EirGenix, Inc. is responsible for development and manufacturing and Sandoz will have the right to commercialize the medicine upon approval in all markets excluding China and Taiwan. On December 20, 2021, Sandoz announced submission of a Biologics License Application for a proposed biosimilar trastuzumab (150 mg) to the US Food and Drug Administration.

Sandoz has been developing and providing oncology medicines for over 30 years. Today, it has more than 50 such medicines, including chemotherapeutics, biologics, hormones and supportive care treatments, for the treatment of a wide range of cancers. The collaboration with EirGenix, Inc. will enable Sandoz to build on its leading generic and biosimilar oncology portfolio to further expand patient access, while contributing to the sustainability of healthcare systems.