On September 23, 2021 Endo International plc (NASDAQ: ENDP) reported that its subsidiary Endo Ventures Limited has entered into definitive agreements with SK Biopharmaceuticals for the development, registration, supply, commercialization and distribution of cenobamate on an exclusive basis in Canada (Press release, SK biopharmaceuticals, DEC 23, 2021, View Source [SID1234597672]). Paladin Labs Inc., an operating company of Endo, will be responsible for all commercial activities in Canada related to cenobamate.

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"We are very pleased to work with SK Biopharmaceuticals to possibly bring a new treatment option to the market for appropriate Canadian epilepsy patients," said Livio Di Francesco, Vice President and General Manager of Paladin. "This transaction continues to build our neurology franchise. Cenobamate has shown promising clinical evidence to address an unmet need and it has the potential to become a very important addition to our existing Canadian portfolio."

Cenobamate is a novel small molecule with a dual mechanism of action under investigation for treating seizures; it is not approved in Canada. 1, 2,3 Although its precise mechanism of action is unknown, cenobamate, at clinically relevant concentrations, acts both as a positive allosteric modulator of the γ-aminobutyric acid (GABAA) ion channel and inhibits voltage-gated sodium currents.2,3 Long-term data of cenobamate is being studied in the open-label extensions of the double-blind placebo control trials as well as the open-label safety study in adults with uncontrolled focal-onset seizures.4 Additionally, cenobamate is being assessed in an ongoing randomized, double-blind, placebo-controlled trial evaluating its safety and efficacy as adjunctive therapy in patients with primary generalized tonic-clonic seizures (NCT03678753).5

About Cenobamate
Cenobamate was discovered by SK Biopharmaceuticals and SK life science and is an anti-seizure medication for the treatment of partial-onset seizures in adults (also known as focal-onset seizures). In November 2019, the U.S. Food and Drug Administration approved cenobamate tablets, marketed under the trademark XCOPRI (cenobamate tablets) CV in the U.S., for such treatment. In March 2021, the European Commission granted marketing authorization for cenobamate tablets, marketed under the trademark ONTOZRY in Europe, for such treatment.

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 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 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 Aarvik Therapeutics and ArriVent Biopharma Inc. reported a strategic research collaboration to develop and commercialize an undisclosed oncology-focused drug (Press release, ArriVent Biopharma, DEC 23, 2021, https://arrivent.com/aarvik-therapeutics-and-arrivent-biopharma-announce-research-collaboration-to-advance-novel-oncology-drug-candidate-using-aarviks-proprietary-modular-antibody-platform/ [SID1234597656]).

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As part of the collaboration, Aarvik is responsible for the discovery and preclinical development of the novel molecule that is based on its unique modular platform that combines multiple target mechanisms. ArriVent will be responsible for clinical development and commercialization of the collaboration drug candidate that successfully achieves target criteria. For this program, Aarvik will receive an upfront payment and research funding, along with the opportunity for option fees, as well as development and commercial milestones for a potential total value of approximately $100 million, plus royalties.

"Aarvik’s innovative platform enables us to rapidly create a pipeline of differentiated programs across a range of therapeutic targets," said Jagath Reddy Junutula, Ph.D., Co-founder, President and CEO of Aarvik Therapeutics. "This collaboration with ArriVent leverages their strong global development expertise, enabling us to expeditiously advance a novel cancer therapeutic into the clinic. We are excited to partner with ArriVent’s team, which brings a proven clinical track record in this space. Collaborations such as this one will help Aarvik execute its mission to deliver transformative therapeutics to patients in an accelerated timeframe."

"Our main business focus at ArriVent is to identify and secure strategic partnerships with companies such as Aarvik who share a similar vision of advancing highly innovative molecules for difficult to treat diseases such as cancer," said Bing Yao, Ph.D., Chairman, Co-founder and CEO of ArriVent Biopharma. "Developed with their proprietary modular antibody technology, we believe the molecule resulting from this research collaboration will demonstrate enhanced efficacy and safety over available therapies. Coupled with ArriVent’s expertise in global drug development, we aim to rapidly advance this highly novel therapy for patients to benefit globally."