On January 6, 2022 RenovoRx, Inc. (NASDAQ: RNXT), a biopharmaceutical company and innovator in targeted cancer therapy, reported that Shaun Bagai, Chief Executive Officer, will participate in the H.C. Wainwright Bioconnect Virtual Conference on behalf of RenovoRx (Press release, Renovorx, JAN 6, 2022, View Source [SID1234598371]). The conference will be held January 10-13, 2022.

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Mr. Bagai’s investor presentation will be available on-demand beginning January 10th at 7:00 am EST.

Management will be available during the H.C. Wainwright Bioconnect conference for 1-on-1 meetings with the investment community. To schedule a meeting please reach out to your H.C. Wainwright representative or KCSA Strategic Communications by emailing [email protected].

For further information about the H.C. Wainwright Bioconnect conference and to register to attend virtually click here.

A webcast of the event will be available for a limited time on the Events page in the Investors section of the Company’s website at View Source

On January 6, 2022 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported that it has entered into an exclusive license agreement with Gaelan Medical Trade LLC ("Gaelan Medical"), a wholly owned subsidiary of the Ghassan Aboud Group (GAG), for Talicia (omeprazole magnesium, amoxicillin and rifabutin)1, an H. pylori therapy, in the United Arab Emirates (UAE) (Press release, RedHill Biopharma, JAN 6, 2022, View Source [SID1234598388]).

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Under the terms of the agreement, RedHill will receive an upfront payment of $2 million and is eligible for additional milestone payments as well as tiered royalties up to mid-teens on net sales of Talicia in the UAE. Gaelan Medical will receive the exclusive rights to commercialize Talicia in the UAE, as well as a right of first refusal to commercialize Talicia in the Gulf Cooperation Council region (Saudi Arabia, Kuwait, Qatar, Bahrain and Oman) for a pre-determined period.

"We are delighted to partner with Gaelen Medical to help bring Talicia to H. pylori patients in the UAE and potentially other territories in the region," said Dror Ben-Asher, RedHill’s CEO. "This partnership is particularly important given that H. pylori, a major public health concern, impacts up to 84% of the population in the region2 and is one of the strongest risk factors for gastric cancer, leading to a recent regional clinical consensus meeting calling for eradication therapy to be offered to all individuals infected with H. pylori3. We are seeing rapid growth of Talicia in the U.S. in light of the alarming failure rates of clarithromycin-based therapies and growing physician awareness of the need for highly effective first-line H. pylori therapy. We continue to explore with potential partners the expansion of Talicia’s reach into additional ex-U.S. territories."

"H. pylori can cause extensive damage if not properly eradicated first-time and there is considerable need for a therapy like Talicia in the UAE, where 41% of the population4 is affected and have limited options for treatment," said Ghassan Aboud, Chairman of GAG. "Talicia would become the first approved combination product in the UAE specifically designed to treat H. pylori, and we are excited to be partnering with RedHill and at the prospect of realizing Talicia’s potential to help patients with H. pylori infection in the UAE and potentially other territories in the region."

About Talicia
Talicia is the only rifabutin-based therapy approved for the treatment of H. pylori infection and is designed to address the high resistance of H. pylori bacteria seen with other antibiotics. The high rates of H. pylori resistance to clarithromycin have led to significant rates of treatment failure with clarithromycin-based therapies and are a strong public health concern, as highlighted by the ACG, FDA and the World Health Organization (WHO) in recent years.

Talicia is a novel, fixed-dose, all-in-one oral capsule combination of two antibiotics (amoxicillin and rifabutin) and a proton pump inhibitor (PPI) (omeprazole). In November 2019, Talicia was approved by the U.S. FDA for the treatment of H. pylori infection in adults. In the pivotal Phase 3 study, Talicia demonstrated 84% eradication of H. pylori infection in the intent-to-treat (ITT) group vs. 58% in the active comparator arm (p<0.0001). Minimal to zero resistance to rifabutin, a key component of Talicia, was detected in RedHill’s pivotal Phase 3 study. Further, in an analysis of data from this study, it was observed that subjects who were confirmed adherent5 to their therapy had response rates of 90.3% in the Talicia arm vs. 64.7% in the active comparator arm6.

Talicia is eligible for a total of eight years of U.S. market exclusivity under its Qualified Infectious Disease Product (QIDP) designation and is also covered by U.S. patents which extend patent protection until 2034 with additional patents and applications pending and granted in various territories worldwide.

About H. pylori
H. pylori is a bacterial infection that affects approximately 35%7 of the U.S. population, with an estimated two million patients treated annually8. Worldwide, more than 50% of the population has
H. pylori infection, which is classified by the WHO as a Group 1 carcinogen. It remains the strongest known risk factor for gastric cancer9 and a major risk factor for peptic ulcer disease10 and gastric mucosa-associated lymphoid tissue (MALT) lymphoma11. More than 27,000 Americans are diagnosed with gastric cancer annually12. Eradication of H. pylori is becoming increasingly difficult, with current therapies failing in approximately 25-40% of patients who remain H. pylori-positive due to high resistance of H. pylori to antibiotics – especially clarithromycin – which is still commonly used in standard combination therapies13.

On January 6, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapies leveraging CRISPR-based technologies, reported its expected milestones and the following strategic priorities for 2022 (Press release, Intellia Therapeutics, JAN 6, 2022, View Source [SID1234598317]):

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Accelerating clinical validation of in vivo pipeline: Further characterize the safety and efficacy of NTLA-2001, including in patients with cardiomyopathy and complete enrollment of the Phase 1 study; establish the initial clinical profile of NTLA-2002 as a single-dose therapy for the treatment of hereditary angioedema (HAE).
Strategic pipeline expansion: Significantly progress in vivo and ex vivo pipeline, including determining the initial safety profile of NTLA-5001 for acute myeloid leukemia (AML), advancing in vivo insertion candidates and nominating multiple new development candidates.
Platform innovation: Broaden the Company’s industry-leading platform through expansion of Intellia’s genome editing, delivery and cell engineering capabilities.
"Unequivocally, 2021 was a landmark year for Intellia. We demonstrated that our proprietary CRISPR-based platform and LNP technology can turn revolutionary science into potentially transformational medicines. Our platform enables us to advance genome editing approaches, which maximizes our ability to target a multitude of life-threatening diseases," said Intellia President and Chief Executive Officer John Leonard, M.D. "As we begin 2022 with great momentum, we are poised to significantly expand our full-spectrum pipeline of potentially curative therapies with the nomination of at least two new in vivo candidates and our first allogeneic development candidate during the year. Importantly, we look forward to sharing additional data from the ongoing study of NTLA-2001 and interim results from the Phase 1/2 study of NTLA-2002, which we expect will further demonstrate the modularity of our genome editing platform."

Anticipated 2022 Milestones:

In Vivo Programs

NTLA-2001 for ATTR amyloidosis: NTLA-2001 is the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body, and has the potential to be the first single-dose treatment for transthyretin (ATTR) amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc. (Regeneron).
Today, Intellia announced that the first patient in the cardiomyopathy arm of the Phase 1 study has been dosed with NTLA-2001. This follows the Company’s recent announcement that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) approved a protocol amendment for the Company’s ongoing Phase 1 study of NTLA-2001 to include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study now includes patients with ATTR-CM enrolled in new dose-escalation and expansion cohorts. The inclusion of the ATTR-CM patient population is in addition to the original Phase 1 study, which continues to evaluate NTLA-2001 in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Intellia expects to complete enrollment of the Phase 1 study for both ATTRv-PN and ATTR-CM subjects in 2022.
Intellia intends to present additional interim clinical data of NTLA-2001 in ATTRv-PN patients from Part 1, the single-ascending dose portion, and to initiate Part 2, a single-cohort expansion, in the first quarter of 2022. Data to be presented at a company-sponsored event will be from all four ATTRv-PN dose cohorts in Part 1 and include safety and serum TTR knockdown for Cohorts 3 and 4, as well as an early look at durability across all cohorts.
NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and to eliminate the significant treatment burden associated with currently available HAE therapies.
In December 2021, Intellia dosed the first patient in its second clinical study of a CRISPR-based therapeutic candidate evaluating NTLA-2002 for HAE. The first-in-human Phase 1/2 trial is expected to evaluate the safety, tolerability and activity of NTLA-2002 in adults with Type I or Type II HAE, and will continue to leverage insights gained from the development of NTLA-2001.
The Company expects to present interim data from the Phase 1/2 study in the second half of 2022. These results are expected to characterize the emerging safety and activity profile of NTLA-2002 and demonstrate preliminary proof-of-concept.
NTLA-3001 for AATD-associated lung disease: NTLA-3001 is Intellia’s wholly owned CRISPR-mediated in vivo targeted gene insertion development candidate. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to address alpha-1 antitrypsin deficiency (AATD)-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or lung transplant in severe cases.
Intellia is conducting Investigational New Drug (IND)-enabling activities for NTLA-3001 with plans to file an IND or IND-equivalent in 2023. The Company also continues to explore additional editing strategies for AATD.
Ex Vivo Programs

NTLA-5001 for AML: NTLA-5001 is an investigational autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen for the treatment of all genetic subtypes of acute myeloid leukemia (AML).
In the fourth quarter of 2021, Intellia initiated screening of patients in the Phase 1/2a study of NTLA-5001 for patients with AML. The Company expects to dose its first patient in the coming weeks and enroll patients throughout the year. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof-of-concept for its TCR-based platform.
Modular Platform and Pipeline Expansion

Platform Innovation: Intellia is expanding its industry-leading genome editing platform and scientific leadership through editing, delivery and cell engineering innovations that will enable broader in vivo and ex vivo applications.
Intellia plans to advance at least two new in vivo development candidates by the end of 2022.
The Company expects to nominate its first allogeneic ex vivo development candidate by the first half of 2022.
The Company plans to highlight additional advances to its proprietary technology capabilities, including both genome editing and delivery tools, at upcoming scientific conferences in 2022.
Corporate Updates:

In January, Intellia and Kyverna Therapeutics announced a licensing and collaboration agreement for the development of KYV-201, an allogeneic CD19 CART-cell therapy for the treatment of a variety of B cell-mediated autoimmune diseases. Intellia may exercise an option to lead U.S. commercialization for KYV-201 under a co-development and co-commercialization agreement.
In December 2021, Intellia appointed Derek Hicks as Executive Vice President, Chief Business Officer. Mr. Hicks joins Intellia with more than 25 years of combined business, leadership and biotechnology experience, having most recently served as Head of Business Development at Spark Therapeutics.
Cash Position

Intellia ended the fourth quarter of 2021 with approximately $1.1 billion in cash, cash equivalents and marketable securities.
Intellia’s Presentation at the 40th Annual J.P. Morgan Healthcare Conference

Intellia is scheduled to present virtually at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, at 2:15 p.m. ET. A live audio webcast of Intellia’s presentation can be accessed under the Events and Presentations page of the Investors & Media section on the company’s website at www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for at least two weeks following the presentation.

On January 6, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported an update on its business performance, including preliminary net product sales results of INGREZZA (valbenazine) for 2021, and key clinical development milestones for 2022 and 2023 (Press release, Neurocrine Biosciences, JAN 6, 2022, View Source [SID1234598334]). Kevin Gorman, Chief Executive Officer of Neurocrine Biosciences, will discuss these updates as part of a webcast presentation at the 40th Annual J.P. Morgan Healthcare Conference to be held virtually on Monday, January 10 at 11:15 a.m. Eastern Time, followed by a Question and Answer session at approximately 11:35 a.m. Eastern Time.

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Preliminary Fourth Quarter 2021 INGREZZA (valbenazine) Net Product Sales (Unaudited) Highlights

Unaudited preliminary fourth quarter 2021 INGREZZA net sales were approximately $301 million and $296 million on an inventory adjusted basis
Fourth quarter 2021 INGREZZA net sales and total prescriptions grew 25% and 32% respectively vs. fourth quarter of 2020
Quarterly growth driven by record patients on therapy exiting 2021
Commercial expansion to better meet the needs of healthcare professionals across diverse sites of care on track for completion by the end of Q1 2022
The Company plans to provide full-year 2022 INGREZZA sales guidance on its Q4 and FY 2021 Earnings Call on February 11, 2022
"Our fourth quarter and full-year results highlight INGREZZA’s return to growth in a challenging environment. We exited 2021 helping more patients with tardive dyskinesia than ever before. Furthermore, we now have 13 clinical programs in mid-to-late-stage studies which will generate important data readouts over the next two years," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "This year, our priorities are focused on INGREZZA and continuing to advance our broad pipeline. We are uniquely positioned to drive INGREZZA growth and reinvest in our pipeline to develop potential best-in-class medications that are focused on neurological, neuro-endocrine and psychiatric disorders for patients who deserve better treatment options."

Expected Future Milestones and Key Activities

Program

Indication

Milestones / Key Activities

Valbenazine*

(VMAT2 Inhibitor)

Chorea in Huntington Disease

File Supplemental New Drug Application in Second Half (2H) 2022

Adjunctive Treatment of Schizophrenia

Initiate 2nd Registrational Study in 2022; Top-Line Registrational Data in 2023

Dyskinesia Due to Cerebral Palsy

Top-Line Registrational Data in 2023

NBI-827104**

(Selective T-Type CaV

Channel Blocker)

Essential Tremor

Top-Line Phase 2 Data in Mid-2022

Rare Pediatric Epilepsy: CSWS

Top-Line Phase 2 Data in 2H 2022

NBI-1117568†

(Selective M4 Agonist)

Treatment of Schizophrenia

Initiate Phase 2 Study in 2022

New Chemical Entity

Neurological or Psychiatric Indication

Initiate Phase 1 Study in 2022

Crinecerfont

(CRF1 Receptor Antagonist)

Congenital Adrenal Hyperplasia (Adult)

Top-Line Registrational Data in 2023

Congenital Adrenal Hyperplasia (Pediatric)

Top-Line Registrational Data in 2023

NBI-1065845‡

(AMPA Potentiator)

Inadequate Response to Treatment in

Major Depressive Disorder

Phase 2 Data in 2023

NBI-1065846‡

(GPR-139 Agonist)

Anhedonia in Depression

Phase 2 Data in 2023

NBI-921352∝

(Selective NaV1.6 Channel
Blocker)

Focal Onset Seizure in Adults

Phase 2 Data in 2023

Key: VMAT2 = Vesicular Monoamine Transporter 2; CaV = Calcium Channel, Voltage-Gated; CSWS = Epileptic Encephalopathy with Continuous Spike and Wave During Sleep; M4= M4 Muscarinic Receptor; CFR1 = Corticotropin-Releasing Factor Type 1; AMPA = Alpha-Amino-3-Hydroxy-5-Methyl-4-Isoxazole Propionic Acid; GPR = Orphan G Protein Coupled Receptor; NaV1.6 = Sodium Channel, Voltage-Gated

Neurocrine Bioscience Partners: * Mitsubishi Tanabe Pharma Corporation has commercialization rights in East Asia;
** In-Licensed from Idorsia Pharmaceuticals; † In-Licensed from Sosei Group Corporation; ‡ Partnered with Takeda Pharmaceutical Company Limited; ∝ In-Licensed from Xenon Pharmaceuticals

About Tardive Dyskinesia (TD)
Tardive dyskinesia (TD) is an involuntary movement disorder characterized by uncontrollable, abnormal and repetitive movements of the torso, extremities and/or face, which can include hand or foot movements, rocking of the torso, lip smacking, grimacing, tongue protrusion, facial movements or blinking, as well as puckering and pursing of the lips. The condition is associated with taking certain mental health medicines such as antipsychotics, which are commonly prescribed to treat mental illnesses such as bipolar disorder, depression and schizophrenia. In patients with TD, these treatments are thought to result in irregular dopamine signaling in a region of the brain that controls movement. The symptoms of TD can be severe and are often persistent and irreversible. TD is estimated to affect approximately 600,000 people in the U.S.

About INGREZZA (valbenazine) Capsules
INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is an FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. INGREZZA, developed by Neurocrine Biosciences, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with most psychiatric medications such as antipsychotics or antidepressants.

Important Information

Approved Use
INGREZZA (valbenazine) capsules is a prescription medicine used to treat adults with movements in the face, tongue, or other body parts that cannot be controlled (tardive dyskinesia).

It is not known if INGREZZA is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Do not take INGREZZA if you:

are allergic to valbenazine, or any of the ingredients in INGREZZA.
INGREZZA may cause serious side effects, including:

Sleepiness (somnolence). Do not drive, operate heavy machinery, or do other dangerous activities until you know how INGREZZA affects you.
Heart rhythm problems (QT prolongation). INGREZZA may cause a heart problem known as QT prolongation.
Symptoms of QT prolongation may include:
• fast, slow, or irregular heartbeat

• shortness of breath

• dizziness or fainting

Tell your healthcare provider right away if you have a change in your heartbeat (a fast or irregular heartbeat), or if you faint.
Abnormal movements (Parkinson-like). Symptoms include: shaking, body stiffness, trouble moving or walking, or keeping your balance.
Before taking INGREZZA, tell your healthcare provider about all of your medical conditions including if you: have liver or heart problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins and herbal supplements.

The most common side effect of INGREZZA is sleepiness (somnolence). Other side effects
include changes in balance (balance problems, dizziness) or an increased risk of falls, headache, feelings of restlessness, dry mouth, constipation, and blurred vision.

These are not all of the possible side effects of INGREZZA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see accompanying INGREZZA full Product Information.

On January 6, 2022 RayzeBio, Inc., a targeted radiopharmaceutical company developing an innovative pipeline against validated solid tumor targets, reported that Ken Song, M.D., President and CEO of the Company, will be presenting at the 40th Annual J.P. Morgan Healthcare Conference being held virtually on Tuesday, January 11, 2022 (Press release, RayzeBio, JAN 6, 2022, View Source [SID1234598372]).

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J.P. Morgan Healthcare Conference
Date: Tuesday, January 11, 2022
Time: 11:00 a.m. – 11:25 a.m. EST
Please click on the below link to find additional details about the J.P. Morgan Healthcare Conference
View Source