On July 10, 2024 HepaRegeniX GmbH, a clinical stage company developing a novel regenerative therapy for the treatment of acute and chronic liver diseases, reported the closing of a Series C round led by Vesalius Biocapital IV with participation of existing investors Novo Holdings, Boehringer Ingelheim Venture Fund (BIVF), and High-Tech Gründerfonds (HTGF). The new funds of €15 million will be used to advance the clinical development of the Company’s clinical candidate HRX-215.

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As part of the financing, Fabienne Roussel, Partner at Vesalius Biocapital, joins the Non-Executive Board of Directors. Elias Papatheodorou will become Chief Executive Officer, moving from Chair of the Board.

Elias Papatheodorou, Chair of the Board, said: "We are thrilled to secure this significant financing, which underscores the confidence our investors have in our science and our capabilities to bring effective treatments to patients suffering from liver diseases. With the new funds, HepaRegeniX will advance with its clinical plans for a Phase Ib study in the US and an international Phase IIa study to enhance liver recovery and prevent liver failure."

HRX-215 is a small molecule inhibitor of Mitogen-Activated Protein (MAP) Kinase Kinase 4 (MKK4). Inhibition of MKK4 unlocks the regenerative capacity of hepatocytes and can strongly boost liver regeneration in patients. This is highly relevant for patients with liver metastases or primary liver tumors, as resection of the tumors is the only curative therapeutic approach. Liver regeneration is also key in liver transplant and especially in enabling left liver lobe living donor transplant. This strategy could significantly reduce waiting lists for liver transplant candidates. Promising preclinical data have confirmed HRX-215’s capacity for liver regeneration, and HepaRegeniX has successfully completed a Phase I trial. These results have been published in the prestigious journal Cell.

"Vesalius Biocapital is excited to support HepaRegeniX as it progresses into the next phase of clinical development for HRX-215. There is an immense need for a treatment that can induce liver regeneration in patients suffering from liver damage, liver tumors, as well as in transplant settings. HRX-215 has potential to help these patients and make a meaningful impact on their lives. I look forward to working with HepaRegeniX’ excellent leadership team to advance this promising treatment candidate," commented Fabienne Roussel, Partner at Vesalius Biocapital IV.

Importantly, to take HepaRegeniX through the next stages of clinical development and value creation, Dr. Linda Greenbaum is joining HepaRegeniX as Chief Medical Officer (CMO). She brings extensive expertise and experience in clinical development and translational medicine. She recently served as Executive Director, Translational Medicine at Novartis in the US. Before joining Novartis, she held the position of Director of Clinical Development at Janssen R&D and served on the faculty of Thomas Jefferson University and University of Pennsylvania where she led a research laboratory investigating liver regeneration and fibrosis. Dr. Greenbaum holds an MD from the Columbia University Vagelos College of Physicians and Surgeons.

"I am honored to join HepaRegeniX at such a critical juncture in its development," added Dr. Linda Greenbaum, Chief Medical Officer of HepaRegeniX. "MKK4 is a key regulator of liver regeneration, and MKK4 inhibition has been shown to induce liver regeneration after a partial hepatectomy. With this mode of action, HRX-215 has an immense potential to improve outcomes for patients who are currently not able to undergo potentially curative surgical resections due to liver tumors, and other patient groups affected by liver failure. I look forward to working with the talented team at HepaRegeniX to advance the clinical development of HRX-215 through Phase II trials and beyond, with the ultimate goal of improving outcomes for patients with liver diseases worldwide."

Elias Papatheodorou concluded: "It is a pleasure to welcome Dr. Linda Greenbaum to our leadership team. With her extensive knowledge and expertise, she will guide the future clinical development of HRX-215. With our new funding and the extension to our experienced leadership team, we hope to bring a much needed therapy to patients suffering from liver diseases."

(Press release, HepaRegeniX, JUL 10, 2024, View Source [SID1234666459])

On July 10, 2024 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), reported that, the Compensation Committee of Crinetics’ Board of Directors granted non-qualified stock option awards to purchase an aggregate of 137,900 shares of its common stock to eighteen new non-executive employees under the Crinetics Pharmaceuticals, Inc. 2021 Employment Inducement Incentive Award Plan (the "2021 Inducement Plan") (Press release, Crinetics Pharmaceuticals, JUL 10, 2024, View Source [SID1234644790]). The stock options were granted as inducements material to the employees entering into employment with Crinetics in accordance with Nasdaq Listing Rule 5635(c)(4).

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The 2021 Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously employees of Crinetics, or following a bona fide period of non-employment, as an inducement material to such individuals’ entering into employment with Crinetics, pursuant to Nasdaq Listing Rule 5635(c)(4). The options have an exercise price of $50.80 per share, which is equal to the closing price of Crinetics’ common stock on The Nasdaq Global Select Market on July 10, 2024. The shares subject to the stock options will vest over four years, with 25% of the shares vesting on the one-year anniversary of the applicable vesting commencement date and the balance of the shares vesting in a series of 36 successive equal monthly installments thereafter, subject to each employee’s continued employment with Crinetics on such vesting dates. The options are subject to the terms and conditions of the 2021 Inducement Plan and the terms and conditions of a stock option agreement covering the grant.

On July 10, 2024 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases and cancer, reported that the Compensation Committee of the company’s Board of Directors granted one employee a nonqualified stock option to purchase 72,000 shares of its common stock with an exercise price of $0.55 per share, which is equal to the closing price of Kezar’s common stock on July 8, 2024, the grant date of the award (Press release, Kezar Life Sciences, JUL 10, 2024, View Source [SID1234644780]). The stock option was granted as an inducement award material to the individual entering into employment with Kezar, in accordance with Nasdaq Listing Rule 5635(c)(4).

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The stock option will vest over a four-year period, with 25% of each option vesting on the first anniversary of the employee’s start date, and 1/48th of the total shares vesting monthly thereafter, subject to continued employment on each vesting date. The option is subject to the terms and conditions of Kezar’s 2022 Inducement Plan and the stock option agreement covering the grant.

On July 10, 2024 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, reported expected milestones in the second half of calendar 2024 and recapped key achievements year-to-date (Press release, Citius Pharmaceuticals, JUL 10, 2024, View Source [SID1234644779]).

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Leonard Mazur, Chairman and CEO of Citius stated, "Our solid execution since the beginning of the year sets us up for potentially transformative catalysts in the coming months. Mino-Lok’s strong Phase 3 topline results support its potential to become part of the standard of care for treating catheter-related bloodstream infections. Mino-Lok would also have a first and only advantage in a market with no approved or investigational products for salvaging infected central venous catheters. For LYMPHIR, the FDA is currently reviewing our Biologics License Application, with an expected decision on August 13th. We are preparing for near-term commercialization of LYMPHIR if approved. These near-term catalysts should enable the company to optimize its current cash runway, future cash needs, as well as create potential non-dilutive cash opportunities.

"Our primary purpose and mission for these late-stage assets is to provide best-in-class, highly effective treatment options for patients and caregivers," Mazur added. "Ultimately, we believe our achievements and milestones offer powerful levers for value creation."

Late-Stage Assets: Mino-Lok and LYMPHIR

Citius significantly advanced its two late-stage product candidates in the first six months of 2024, and expects several key milestones in the near term.

Mino-Lok: Citius recently announced positive topline data from the Phase 3 study of Mino-Lok in catheter-related bloodstream infections (CRBSIs). Primary and secondary endpoints were met with statistical significance.
Next steps for the Mino-Lok program are to prepare a submission to the U.S. Food and Drug Administration (FDA) and schedule a Type B meeting.
LYMPHIR: In March 2024, the FDA accepted the Company’s Biologics License Application (BLA) for LYMPHIR (denileukin diftitox), an IL-2-based immunotherapy for the treatment of patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL).
A decision on the LYMPHIR BLA is expected on August 13, 2024, the FDA’s assigned Prescription Drug User Fee Act (PDUFA) action date. If approved, Citius is preparing for LYMPHIR commercialization in 2024.
Considered a new biologic by the FDA, LYMPHIR would potentially be eligible for 12 years of exclusivity, if approved.
In addition to the initial indication, two investigator-initiated Phase 1 studies of LYMPHIR are underway to explore how the differentiated mechanism-of-action may offer potential indications beyond CTCL. The studies are in progress at the University of Pittsburgh and the University of Minnesota.
Specialty Pharma Asset: Halo-Lido

An end of Phase 2b trial meeting with the FDA was held to discuss the positive data for Halo-Lido, a prescription strength topical for symptomatic hemorrhoid treatment. The data showed a meaningful reduction in symptom severity when compared to individual components alone.
Continuing engagement with the FDA will guide the Company’s next phase of development for Halo-Lido.
Operational Milestones

Operational achievements by Citius in the first six months of 2024 delivered additional capital and stronger levers for value creation.

Citius took steps to strengthen its capital structure in preparation for LYMPHIR commercialization, if approved. A $15 million registered direct offering was completed in April 2024, extending the Company’s cash runway.
Citius plans to merge a wholly owned subsidiary with TenX Keane Acquisition (Nasdaq: TENK) to form publicly listed company, Citius Oncology, Inc. The transaction is pending review by the U.S. Securities and Exchange Commission (SEC) and TENK shareholder approval as well as contractual and customary closing conditions.
About Mino-Lok

Mino-Lok (MLT), a novel antibiotic lock solution that combines minocycline, ethanol and edetate disodium, is designed to treat patients with catheter-related blood stream infections. Citius licensed Mino-Lok from an affiliate of The University of Texas MD Anderson Cancer Center. Mino-Lok is designed to offer an alternative to removing and replacing a central venous catheter (CVC), which may lead to a reduction in serious adverse events and cost savings to the healthcare system. If approved, Mino-Lok would be the first and only FDA-approved treatment that salvages central venous catheters that cause central line-related blood stream infections.

About LYMPHIR (denileukin diftitox-cxdl)

LYMPHIR is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. In 2011 and 2013, the FDA granted orphan drug designation to LYMPHIR for the treatment of PTCL and CTCL, respectively. In 2021, denileukin diftitox received regulatory approval in Japan for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). Subsequently in 2021, Citius acquired an exclusive license with rights to develop and commercialize LYMPHIR in all markets except for Japan and certain parts of Asia.

About Halo-Lido

Halo-Lido (CITI-002) is a proprietary topical formulation intended to provide symptomatic relief to individuals suffering from hemorrhoids. Hemorrhoids are a gastrointestinal disorder characterized by pain, swelling, itching, tenderness, and bleeding. Although hemorrhoids are not life-threatening, individual patients often suffer painful symptoms that can limit social activities and have a negative impact on the quality of life. More than half of the U.S. population will experience hemorrhoidal disease at least once in their life. Each year, nearly 10 million patients in the U.S. report symptoms.

On July 10, 2024 Rgenta Therapeutics, a biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported the clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA) for RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML) (Press release, Rgenta Therapeutics, JUL 10, 2024, View Source [SID1234644778]).

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"Clearance of our first IND application is a significant milestone in Rgenta’s mission to develop oral, small molecule RNA-targeting medicines to treat previously incurable diseases," said Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta. "We look forward to initiating clinical studies of RGT-61159 with a first-in-human Phase1a/1b clinical study in adults with ACC and CRC to potentially provide a new therapeutic option for patients with these difficult-to-treat cancers."

"Development of small molecule drugs targeting oncogenic drivers such as MYB, has proven challenging in the past," said Travis Wager, Ph.D., co-founder, president and chief scientific officer. "RGT-61159 demonstrates potent inhibition of oncogenic MYB protein production and significant inhibition of tumor growth at tolerated doses in preclinical models of ACC and other cancers, and we are excited to move this novel therapeutic into clinical evaluation."

About RGT-61159
RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB resulting in the inhibition of oncogenic MYB protein production, which has the potential to inhibit proliferation or induce cell death of cancer cells that overexpress MYB protein. MYB acts as a master regulator of cell proliferation differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemias (AML), T-cell acute lymphoblastic leukemias (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer. Additional information on the planned Phase 1a/1b clinical trial can be accessed at clinicaltrials.gov (NCT06462183).

About Adenoid Cystic Carcinoma (ACC)
It is estimated that approximately 200,000 people are living with ACC throughout the world including 11,000 in the US. While it is a rare cancer, ACC is the second most common cancer type arising in the salivary gland and is an aggressive malignancy with a tendency to infiltrate surrounding nerves and metastasize to distant sites. Overactivation of the MYB oncogene has been described as a hallmark of ACC and is noted in over 90% of ACC. Treatment for ACC is extremely challenging and may include surgery and/or radiation, which often fails to control local tumor recurrence and distant metastases. There are no effective targeted therapies available for patients with recurrent and/or metastatic disease. There is thus an unmet medical need for new therapeutic targets and treatment strategies for patients with this fatal cancer.

About Colorectal Cancer (CRC)
CRC is the third most prevalent cancer and the second leading cause of cancer-related mortality worldwide. According to the World Health Organization, in 2022, more than 1.9 million cases of CRC were diagnosed. Despite the improved early detection of CRC and the recent success of targeted therapeutics, approximately 15%-30% of patients present with metastases and 20%-50% of patients with initially localized disease will develop metastases. Patients with relapsed or refractory CRC who have exhausted all the available standard of care therapy options, have a very poor prognosis. MYB is significantly overexpressed in 80-85% of CRC and has been frequently found to be a predictive biomarker of tumor aggressiveness and poor prognosis. Developing novel therapies to treat patients with metastatic CRC remains a major unmet medical need.