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.

On July 10, 2024 Therapeutic delivery platform Granza Bio reported the closing of its oversubscribed $7.14M Seed led by Felicis and Refactor, along with Y Combinator and notable angel investors (Press release, Granza Bio, JUL 10, 2024, View Source [SID1234644777]).

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Founded in 2024 by a team of cancer and immunology researchers at the University of Oxford, Granza Bio utilizes the discovery of "attack particles," a powerful suite of weapons in the immune system’s arsenal that can fight diseases such as cancers, autoimmunity and infections.

"With attack particles, we discovered a new feature of the immune system that has the potential to transform the therapeutic landscape. They offer the power of cellular therapies with the off-the-shelf ease of antibody-based drugs," said Granza Bio co-founder Michael Dustin, Professor of Molecular Immunology at Oxford.

However, the team realized they lacked an effective delivery system for packaging and delivering attack particles to specific tissues.

To solve this problem, Granza Bio’s platform offers a unique delivery system that focuses on three key areas:

1) The ability to deliver a variety of cargo types (such as RNAs, proteins, and attack particles)
2) The ability to deliver to a range of tissue sites
3) Overcoming immunological activation triggered by the delivery vehicle

"We are developing a series of precision delivery shells that can encompass and direct any therapeutic cargo to the correct destination in our body. This work will be fundamental for turning promising discoveries in genetic medicine and cancer therapeutics into impactful treatments for patients," said Granza co-founder and Chief Executive Officer, Dr. Ashwin Nandakumar.

"Through the development of a comprehensive library of engineered shells, we aim to tackle the challenges of tropism, immunogenicity, and stability head-on. Our strength lies in the interdisciplinary nature of our team, spanning fundamental immunology to industrial manufacturing and scale-up processes. This is a pivotal moment for the field of drug delivery therapeutics and we are at the forefront of this transformation," said Dr. Ashwin Jainarayanan, Granza Bio’s Chief Scientific Officer.

Dr. Nandakumar, CEO, holds a PhD in Oncology, has previously set up clinical trials, and brings oncology start-up experience. Dr. Jainarayanan, CSO, is a PhD in Interdisciplinary Bioscience and was recently selected as a Schmidt Science Fellow. Prof. Dustin is also the director of research at the Kennedy Institute of Rheumatology at Oxford.

"The immune system is nature’s most powerful mechanism for biological remediation. By improving the delivery and specificity of naturally occurring attack particles, Granza Bio can unlock a new world of capabilities for oncological and immunological diseases," said Tobi Coker, Deal Partner at Felicis. "Granza encompasses a unique combination of significant scientific advancements, a founding team with complementary skills, and deep domain expertise. We are thrilled to be working with them."

"I focus on finding technical founders who are experts in their fields and are magnets for customers, talent, and investors. Both Ashwins really fit that perfectly, in my opinion, as they build a better delivery mechanism for a variety of therapeutics," said Zal Bilimoria, founding partner at Refactor.

The investment round also includes participation from other investors including Metaplanet, Zeno Ventures, Ritual Capital, Pioneer Fund, Oxford Angel Fund, and North South Ventures. Notable angels joined the round including Richard Aberman (former YC Visiting Group Partner), JJ Fliegelman (former YC Visiting Group Partner), Eric Migicovsky (former YC Group Partner), Eric Eldon (former editor of TechCrunch), Eli Brown (founder, Guilded), Eoghan Mccabe (founder, Intercom), Benjamin Bryant (co-founder, Earbits), Yotam Rosenbaum (co-founder, Earbits), Will Olsen (co-founder, Engage Bio) and Joel Meyer.

On July 10, 2024 Lantern Pharma (NASDAQ: LTRN), a clinical-stage biopharmaceutical company leveraging artificial intelligence (AI) and machine learning to transform the cost, pace, and timeline of oncology drug discovery and development, reported a significant advancement towards the development of a diagnostic for its drug candidate LP-184 (Press release, Lantern Pharma, JUL 10, 2024, View Source [SID1234644776]). The diagnostic is currently based on qRT-PCR (quantitative real-time polymerase chain reaction) technology and is focused on quantifying the amount of PTGR1 RNA in patient tumor samples to assess the potential for sensitivity to Lantern’s drug candidate LP-184. The company plans to further develop and validate the assay for its use as a potential tool for patient selection in later stage clinical trials across a broad range of solid tumors that have shown sensitivity to LP-184.

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Lantern has successfully confirmed PTGR1 as a key biomarker that it intends to use to optimize patient selection based on potential tumor sensitivity to the drug candidate LP-184. LP-184 is a precision oncology drug candidate with the potential to address multiple solid tumors. This confirmation marks a crucial step towards the development of a companion diagnostic and potential stratification tool to assist with targeted patient selection. Lantern plans on further validations and development of the assay using qRT-PCR and partnering with central labs and cancer centers for eventual use in patient selection and stratification. In a key publication on the utility and value of biomarkers in oncology trials among some of the most common cancers, titled Does biomarker use in oncology improve clinical trial failure risk? A large‐scale analysis by Parker, et al., 2021 in Cancer Medicine found success of clinical trials to be significantly correlated to the incorporation of biomarkers. In particular, the Parker, et al. publication stated that:

"…Our overall analysis of these four cancers, independent of indication, revealed a fivefold benefit of hazard ratios from the Markov models, suggesting a substantial benefit from biomarker use. The hazard ratio analysis of the Markov biomarker models examined how likely clinical trial success was associated with biomarker use versus no biomarker use. Hazard ratios indicated that for biomarker‐based drugs clinical trial success was largest for breast cancer (12‐fold) followed by melanoma (eightfold) and lung cancer (sevenfold) …Our data provide the most extensive look at biomarker use to date in oncology, with an advanced statistical method. Our findings indicate that biomarkers provide a statistically significant benefit, despite the fact our study includes biomarkers not yet FDA approved."

By incorporating the PTGR1 biomarker into LP-184’s development strategy, Lantern Pharma is aligning with best practices in precision medicine and aiming to increase the likelihood of successful clinical outcomes in future clinical trials. PTGR1 levels have been measured to be higher in certain cancer cells than in normal cells, and Lantern is leveraging this biological activity to target the cancer indications believed most likely to respond to drug candidate LP-184. In the October 2023 paper in Molecular Cancer Therapeutics, Lantern along with collaborators from Fox Chase Cancer Center published clear evidence that higher potency of LP-184 (measured in IC50 values) was directly correlated with higher expressions of PTGR1 and that cancer cell lines that did not have PTGR1 expression remained stable in the presence of LP-184 (see figure 1).

"This milestone represents a significant leap forward in our precision oncology approach and in ensuring that we enrich our future LP-184 clinical trials with the patients we believe will be most likely to benefit," said Panna Sharma, CEO of Lantern Pharma. "By working to develop a companion diagnostic for LP-184, we’re not just advancing a drug candidate; we’re paving the way for more personalized and effective cancer treatments for patients that have the highest likelihood of benefitting from the therapy. The planned use of biomarkers like PTGR1 in our clinical trials exemplifies our commitment to data-driven, patient-centric drug development."

These steps toward development of this companion diagnostic align with Lantern Pharma’s commitment to leveraging cutting-edge technology in drug development. By combining AI-driven insights with advanced diagnostic tools, the company aims to accelerate the drug development process and improve patient outcomes.

Lantern Pharma plans to implement this assay in upcoming clinical trials for LP-184, potentially streamlining the development process and increasing the likelihood of successful outcomes. LP-184— a novel therapeutic in clinical development for the potential treatment of malignant gliomas, pancreatic cancer, and atypical teratoid rhabdoid tumors (ATRT)— has also been granted an Orphan Drug Designation by the FDA, along with a Rare Pediatric Disease Designation.