On December 23, 2021 Amgen (NASDAQ:AMGN) reported that it will present at the Goldman Sachs 14th Annual Healthcare CEOs Unscripted Conference at 1:00 p.m. ET on Thursday, Jan. 6, 2022 (Press release, Amgen, DEC 23, 2021, View Source [SID1234597657]). Robert A. Bradway, chairman and chief executive officer at Amgen will present at the conference. Live audio of the conference call will be broadcast over the internet simultaneously and will be available to members of the news media, investors and the general public.

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The webcast, as with other selected presentations regarding developments in Amgen’s business given at certain investor and medical conferences, can be accessed on Amgen’s website, www.amgen.com, under Investors. Information regarding presentation times, webcast availability and webcast links are noted on Amgen’s Investor Relations Events Calendar. The webcast will be archived and available for replay for at least 90 days after the event.

On December 23, 2021 Lion TCR Pte Ltd reported that it has received Fast Track Designation from United States Food and Drug Administration (U.S. FDA) for LioCyx-M004, autologous T-cells transfected with mRNA encoding Hepatitis B surface antigen (HBsAg) specific TCR, for the treatment of HBV-related hepatocellular carcinoma (HBV-related HCC) (Press release, Lion TCR, DEC 23, 2021, View Source [SID1234597683]). This Fast Track Designation provides Lion TCR with an expedited path towards the regulatory approval for its leading investigational product, LioCyx-M004, which is being developed as a potential first-in-class drug for HBV-related HCC. This designation was granted based on that the efficacy of LioCyx-M004, as demonstrated by an improvement in the overall survival in patients with HBsAg-positive HCC relapsed or refractory to prior systemic treatment.

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The FDA Fast Track Designation is designed for sponsors to gain access to expedited drug approval, via eligibility for Accelerated Approval and Priority Review, for medical conditions that are serious and potentially life-threatening, and where there is an unmet medical need through early and frequent meetings with the FDA to discuss drug development plans[1]. The purpose is to get important new drugs to the patient earlier.

In earlier phase 1 study, LioCyx-M004 has showed the well-tolerated safety profile and promising prolonged overall survival. In September 2021, FDA Investigational New Drug (IND) Clearance for a Phase 1b/2 multi-center study has been obtained[2]. This is the first ongoing clinical trial that uses HBV-specific TCR T cell therapy to target HBV-related HCC.

"HBV-related HCC occurs in over 420,000 people every year worldwide and majority of advanced HCC patients relapse quickly after initial treatment. However, existing treatments are very limited especially on improving overall survival. We believe that our innovative TCR-T therapy can fill this urgent and important unmet medical needs. With this Fast Track designation, we look forward to having more frequent communication with the Agency in the hope to attain a more expedited drug approval for our product for patient access. Efforts for patient recruitment for the Phase 1b/2 study in the U.S. and Asia are underway." said Dr Tina Tingting Wang, COO and CMO of Lion TCR.

"The field of T-cell therapy is highly dynamic and competitive. Innovative therapies are released faster than ever through expediated programs like Fast Track. Together with our Orphan Drug Designation obtained for the use of HBV-specific TCR T cell therapy in HCC, we believe this Fast Track approval can drive forward the accelerated regulatory approval our proprietary first-in-class TCR T cell therapy." said Dr Peng Xiaoming, CEO of Lion TCR.

References

U.S. Food and Drug Administration (FDA). Fast Track. Available from: View Source
View Source

On December 23, 2021 Labcorp (NYSE: LH), a leading global life sciences company, reported that it has entered into a definitive agreement to acquire Personal Genome Diagnostics Inc. (PGDx), a leader in cancer genomics with a portfolio of comprehensive liquid biopsy and tissue-based products (Press release, LabCorp, DEC 23, 2021, View Source [SID1234597658]).

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The addition of PGDx and its technology complements and accelerates Labcorp’s existing liquid biopsy capabilities and expands Labcorp’s leading oncology portfolio of next-generation sequencing (NGS)-based genomic profiling capabilities, positioning Labcorp at the forefront of driving better patient outcomes in oncology. PGDx enhances Labcorp’s ability to increase access to oncology care in the global community through kitted solutions that allow hospital systems and laboratories to run these tests internally, bringing precision diagnostics closer to the patient and helping close a common gap in the delivery of cancer care. Labcorp’s global reach also provides the opportunity to bring this technology to pharmaceutical companies for clinical trial research to advance cancer treatments, potentially impacting clinical outcomes in millions of people with cancer.

"Labcorp’s leadership and scale in diagnostic testing and drug development, coupled with PGDx’s innovative technology and suite of capabilities, will accelerate access to personalized treatments for cancer patients globally," said Adam Schechter, chairman and CEO of Labcorp. "PGDx’s comprehensive portfolio of next-generation sequencing products will meaningfully add to our breadth of capabilities, in line with our strategic priority to lead in oncology. PGDx’s technology is well positioned in an important segment with strong growth prospects. We look forward to welcoming PGDx’s talented team and working together to bring world-class diagnostics, technology and treatments within reach for all."

Under the terms of the agreement, Labcorp will pay $450 million in cash at closing and up to an additional $125 million on achieving future performance milestones.

PGDx’s centralized and decentralized offerings will enhance the scalability of the technology and support long-term growth across Labcorp’s oncology portfolio, while also enabling Labcorp to seamlessly offer oncology testing at every stage of care. PGDx offers the only diagnostic kit cleared by the U.S. Food and Drug Administration for pan-solid cancer comprehensive tumor profiling using a 500+ gene panel.

Next-generation sequencing, including liquid biopsy, represents the future of treatment and response monitoring in people with cancer. Liquid biopsy testing can also eliminate the need for an invasive biopsy procedure, reducing costs and improving patient outcomes. When combined with cutting-edge data analysis capabilities, NGS is the most advanced technology in the field today for identifying the best therapy available for each patient. As more cancer patients gain access to NGS testing, Labcorp will be able to offer enriched, actionable and data-driven insights. These insights can be used by pharmaceutical companies and cancer care teams to accelerate patient recruitment for clinical trials and identify patients eligible for approved treatments.

Overall demand for noninvasive tumor profiling and therapeutic response monitoring is expected to grow significantly—largely attributable to technological advances in the identification of biomarkers utilizing NGS, applicability to companion diagnostics and immuno-oncology solutions, and widespread government support.

"We share Labcorp’s vision of improving health care decisions and outcomes through science, data and a continued commitment to innovation," said Megan Bailey, CEO of PGDx. "For over a decade, PGDx has made great progress toward that goal. As a part of the Labcorp family, we have an incredible opportunity to broaden and accelerate our impact on cancer care through Labcorp’s global reach."

The acquisition of PGDx is the latest development in Labcorp’s long-standing commitment to integrate precision medicine into its comprehensive offering of oncology solutions. Labcorp currently offers OmniSeq INSIGHTSM, a NGS-based, precision medicine test for solid tumors, IntelliGEN Myeloid, a NGS-based precision medicine test for myeloid malignancies, and hereditary cancer genetic testing through VistaSeqSM. Labcorp also offers clonoSEQ, the first and only FDA-cleared assay for measurable, residual disease detection, and Resolution ctDx Lung, a non-invasive test for patients with non-small-cell lung cancer.

PGDx 2021 revenues are expected to be approximately $22 million and projected revenues for 2022 are expected to grow to nearly $40 million. The acquisition is expected to be slightly dilutive to Labcorp’s adjusted earnings per share over the next couple of years and provide returns in excess of its cost of capital by year five.

The transaction is subject to customary closing conditions and regulatory approvals, including under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended. The transaction is expected to close in the first half of 2022. Hogan Lovells and Kilpatrick Townsend acted as legal advisors to Labcorp. Cooley LLP acted as legal advisor and Cowen acted as financial advisor to PGDx.

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