On June 5, 2024 Mural Oncology plc (Nasdaq: MURA), a clinical-stage immuno-oncology company developing novel, investigational engineered cytokine therapies designed to address areas of unmet need for patients with a variety of cancers, reported that Caroline Loew, Ph.D., CEO, will present at the East Coast IDEAS Investor Conference on June 13 at 9:15 a.m. EST (Press release, Mural Oncology, JUN 5, 2024, View Source;1-investor-meetings-14th-annual-east [SID1234644128]). A webcast will be available at www.threepartadvisors.com/east-coast and ir.muraloncology.com.

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About IDEAS Investor Conferences

The mission of the IDEAS Conferences is to provide independent regional venues for quality companies to present their investment merits to an influential audience of investment professionals. Unlike traditional bank-sponsored events, IDEAS Investor Conferences are "SPONSORED BY INVESTORS. FOR INVESTORS" and for the benefit of regional investment communities. Conference sponsors collectively have more than $200 billion in assets under management and include: 1102 Partners, Adirondack Research and Management, Allianz Global Investors: NFJ Investment Group, Ariel Investments, Aristotle Capital Boston, Ascend Wealth Advisors, Barrow Hanley Mewhinney & Strauss, BMO Global Asset Management, Constitution Research & Management, Inc., Diamond Hill, First Wilshire Securities Management, Inc., Granahan Investment Management, Great Lakes Advisors, Greenbrier Partners Capital Management, LLC, Hodges Capital Management, Ironwood Investment Management, Keeley Teton Advisors, Luther King Capital Management, Marble Harbor Investment Counsel, North Star Investment Management, Perritt Capital Management, Punch & Associates, Shepherd Kaplan Krochuk, Westwood Holdings Group, Inc., and William Harris Investors. The IDEAS Investor Conferences are held annually and are produced by Three Part Advisors, LLC. Additional information about the events can be located at View Source

On June 5, 2024 Model Medicines, a leading human health company specializing in generative AI-driven drug discovery, reported the nomination of preclinical candidate MDL-4101, a novel-acting small molecule inhibitor of bromodomain-containing protein 4 (BRD4), for the treatment of thyroid and other cancers (Press release, Model Medicines, JUN 5, 2024, View Source [SID1234644127]). MDL-4101 demonstrates the company’s commitment to leveraging AI to discover novel therapies for aggressive cancers with high unmet medical needs. In particular, MDL-4101 was discovered and the BRD4 program [4] was launched to overcome the limitations of previous BRD4 therapeutics, which have failed to reach the pharmacy [5].

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The nomination of MDL-4101 resulted from Model Medicines’ proprietary AI-driven drug discovery platform, GALILEO, modeling the epigenetic oncology target BRD4, a promising but difficult therapeutic target. BRD4, a member of the bromodomain and extra-terminal (BET) protein family, has emerged as a highly validated therapeutic target across a wide range of cancers. As a key regulator of oncogene expression, BRD4 has been shown to drive tumorigenesis in multiple solid and hematologic malignancies, including breast cancer, prostate cancer, leukemias, and lymphomas. Despite this broad potential, BRD4 has proven challenging to drug selectively, leading to safety and tolerability issues, and has been labeled as difficult to drug, or undruggable, due to the inability of conventional approaches to yield selective and tolerable inhibitors. To date, no selective BRD4 inhibitor has received regulatory approval, underscoring the urgent need for new modalities to effectively target this high-value protein [6-10].

Model Medicines deployed its proprietary GALILEO platform to discover and design MDL-4101 as a selective BRD4 inhibitor that could overcome the limitations of previous programs. By leveraging machine learning to explore vast chemical space and predict drug-like properties, Model Medicines successfully identified MDL-4101 as a potent, selective, and orally bioavailable BRD4 inhibitor, succeeding where traditional approaches have struggled. MDL-4101 demonstrates BRD4 binding in cell free assay [11] and robust anti-tumor activity in a preclinical model of thyroid cancer (CGTH-W-1)[12], suppressing cell proliferation and metastatic capacity. Additionally, preclinical studies have shown evidence of activity in human glioblastoma, prostate, and testicular cancers.

"The successful nomination of MDL-4101 as our first preclinical oncology candidate is a significant milestone for Model Medicines and underscores the unparalleled ability of our AI platform to solve previously intractable challenges in drug discovery," said Daniel Haders, Ph.D., CEO and founder of Model Medicines. "BRD4 is a target that has long captivated drug hunters due to its central role in cancer, but has evaded their grasp. By leveraging AI to intelligently discover and design compounds with optimal properties, we discovered a molecule in MDL-4101 that potentially unlocks the full potential of BRD4 inhibition. We believe this is just the beginning of what our AI-powered approach can achieve against undruggable targets in oncology and beyond."

Thyroid cancer is the most common endocrine malignancy and the tenth most common cancer in the world, accounting for an estimated 586,000 cases worldwide in 2020 [13]. Thyroid cancer is also the fifth most common cancer type in the US, with 44,000 cases in 2022 [13, 14]. BRD4 is found to be up-regulated across many human thyroid cancers and cancer models [15]. The most common subtypes include the differentiated thyroid cancers papillary thyroid carcinoma (PTC) and follicular thyroid carcinoma (FTC), which have good prognosis with early intervention [16]. However, treatment options for more aggressive forms, such as anaplastic thyroid carcinoma (ATC) and subtypes such as squamous cell carcinoma of the thyroid (SCCT), remain limited and challenging. Today’s reported results evaluated MDL-4101 in a BRD4-enriched human thyroid cancer model (CGTH-W-1) that was derived from a sample of metastatic SCCT, which has a five-year survival rate of only 6.4% [17]. SCCT is a very aggressive tumor with a poor prognosis. The most recommended treatment involves surgical resection with adjuvant radiotherapy and chemotherapy despite its poor reported outcomes​ [18]. Thus, there is a high unmet medical need for novel therapeutic interventions.

"The preclinical data for MDL-4101 in human thyroid cancer is highly encouraging and speaks to the immense promise of a selectivBRD4 inhibitor," said Dr. Launa Aspeslet, Senior Scientific & Clinical Advisor to Model Medicines. "By unleashing the untapped potential of BRD4 inhibition, we believe MDL-4101 could represent a transformative advance for patients with thyroid and other BRD4 related cancers who currently face a paucity of effective treatment options. We are excited to rapidly progress MDL-4101 into first-in-human studies, while exploring its potential in other cancers driven by BRD4."

Model Medicines plans to initiate Investigational New Drug (IND) enabling studies for MDL-4101, with the goal of submitting an IND application to the U.S. Food and Drug Administration (FDA) and initiating a Phase 1 clinical trial in patients in the near future.

On June 5, 2024 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that Robert M. Davis, chairman and chief executive officer, and Dr. Dean Y. Li, executive vice president and president, Merck Research Laboratories, are scheduled to participate in a fireside chat at the Goldman Sachs 45th Annual Global Healthcare Conference on Tuesday, June 11, 2024, at 10:00 a.m. EDT (Press release, Merck & Co, JUN 5, 2024, View Source [SID1234644126]).

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Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at this weblink.

On June 5, 2024 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson’s disease ("PD"), Parkinson’s-related disorders and other diseases of the Abelson Tyrosine Kinases, reported expansion of its therapeutic pipeline and multiple updates to its Research and Development programs.

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"At Inhibikase we have let fundamental scientific discoveries drive identification of new product candidates that could transform the lives of patients. We first began clinical development of our product candidates in 2021. In 2025, we anticipate having multiple late-stage ready assets across our therapeutic pipeline," commented Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. "The 201 Trial in untreated Parkinson’s disease has reached 94% enrollment and we anticipate enrolling the last patient in mid-June. In parallel, we are seeking grant funding for the 202 trial in Multiple System Atrophy through The National Institute of Neurological Diseases and Stroke (NINDS). Finally, following our pre-IND meeting with the U.S. Food and Drug Administration (FDA) in April, 2024, we have made the decision to redirect our efforts with IkT-001Pro into cardiopulmonary disease, opening a new therapeutic area for the Company. Taken together, we believe we are on track to advance our clinical assets into late-stage trials in the coming year."

Upcoming Milestones and Strategic Updates:

Complete The 201 Trial in untreated Parkinson’s disease. The Company anticipates that the last patient will complete the 12-week treatment period before the close of the third quarter of 2024. The Company expects to report biomarker and outcome data to support the Company’s pursuit of an End of Phase 2 and Phase 3 protocol discussion with the FDA by the end of 2024.

Expansion into cardiopulmonary disease. Following the Company’s pre-IND meeting with the FDA, the Company will submit its IND application to the FDA for IkT-001Pro as a treatment for Pulmonary Arterial Hypertension (PAH) early in the third quarter of 2024, opening a new therapeutic area for the Company. The active ingredient in IkT-001Pro, imatinib, has previously been shown to be disease-modifying for PAH. The Company believes that 001Pro could have a more favorable safety and tolerability profile compared to imatinib for this indication. If approved, IkT-001Pro could be a branded product with all the value drivers of a novel treatment for indication of high unmet need. The IND for 001Pro in PAH represents the seventh the Company has filed since 2019.

Scaling manufacturing of IkT-001Pro. Following the Company’s pre-NDA meeting with the FDA in January, 2024, the Company is scaling its process development efforts for IkT-001Pro to support late-stage clinical development and NDA batch requirements. These activities include development of new dosage forms to differentiate 001Pro tablets from generic imatinib mesylate in alignment with FDA feedback.

Seeking support for the 202 Trial in Multiple System Atrophy through the Other Transaction Authority of NINDS. The National Institute of Neurological Diseases and Stroke (NINDS) is initiating a new funding mechanism for clinical development in neuroscience beginning June, 2024. Through this new program, termed the Other Transaction Authority (OTA), the Company is seeking to support its Phase 2 ‘202 Trial’ in MSA trial using a dedicated U.S. trial network set-up by the Institute.
Discontinuing antiviral development for Progressive Multifocal Leukoencephalopathy (PML): As part of the Company’s strategy to focus on late-stage clinical assets in neurodegeneration, cancer and cardiopulmonary disease, Inhibikase will discontinue development of treatments for PML.

On June 5, 2024 ImmuPharma PLC (LSE:IMM), ("ImmuPharma" or the "Company"), the specialist drug discovery and development company, reported its Final Results for the twelve months ended 31 December 2023 (the "Period") (Press release, ImmuPharma, JUN 5, 2024, View Source [SID1234644124]).

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Key Highlights (including post Period review)

Financials

Loss for the Period of £2.9m (2022: £3.8m)
Research and development expenses of £2.0m (2022: £2.0m)
Administrative expenses of £1.0m (2022: £0.8m)
Share based expense of £0.14m (2022: £0.16m)
Cash balance of £0.2m at 31 December 2023 (31 December 2022: £0.7m)
Lanstead derivative financial asset of £0.6m (2022: £0.3m)
Basic and diluted loss per share of 0.81p ( 2022: 1.26p)
Incanthera financial asset: shares of £0.6m (2022: £0.7m) – warrants of £1 (2022: £1k)
Fundraising in September 2023, comprising gross proceeds of £130,683 in addition to £1.35 million being raised in a Subscription and Direct Subscription
Portfolio

SLE/Lupus

A Phase 3 dose-range study of P140, rather than a Phase 2/3 adaptive study, is the preferred design.
Importantly, the direct Phase 3 route is faster to filing for approval whilst also incorporating the FDA’s request for demonstration of a dose-ranging in the pivotal program
The international SLE Phase 3 dose-range study design and protocol is substantially different from the previous Phase 3 clinical trial completed by ImmuPharma in 2018
Dosing will be significantly higher and subcutaneous injection, once a month, will be administered with a highly convenient and patient friendly autoinjector. The doses are safe and well tolerated.
Two planned interim analyses during the study will allow early detection of the effectiveness of P140
Simbec-Orion appointed as the Contract Research Organisation ("CRO")
CIDP

In May 2023, ImmuPharma received positive guidance from FDA following the PIND meeting that confirms the route for a Phase 2/3 adaptive clinical study of P140 in CIDP
This will be the first pivotal stage study of P140 in patients with CIDP: a rare neurological disease with high medical need
An IND application is now close to submission to the FDA, incorporating all guidance points
An application for Orphan Drug status for CIDP will be also submitted in parallel
Simbec-Orion, has been appointed as the CRO for this program
P140 technology platform

Recent further insights into P140’s mechanism of action ("MOA") confirms its position as the only non-immunosuppressing molecule in clinical development in the industry
The favourable impact of P140 on immune system homeostasis also support P140 as a new potential standard of care not only for SLE sufferers, but for patients suffering from a multitude of autoimmune diseases that are caused by the same underlying malfunction
In April 2024, the Company announced that it has initiated a new intellectual property strategy to significantly enhance the patent life and commercial value for its P140 technology platform
Anti-infectives | Bio-AMB

After multiple in vivo studies assessing the Pharmacokinetic/Pharmacodynamic ("PK/PD") and safety profile of BioAMB, the dose-effect relationship has now been assessed in Part 1 of a new dose-range pharmacodynamic study in an aspergillosis rat model. Part 1 has now been completed – no toxicity related to BioAMB was observed at the active dose
Part 2 of the study will further evaluate the safety of BioAMB at the active dose and confirm the advantage of BioAMB over the other forms of AMB
Cancer

In March 2023 a collaboration with Orano SA on ImmuPharma’s peptide technology was established
Partnering

Active discussions are ongoing with new potential corporate partners across the P140 platform and anti-infective programs.
Corporate

In August 2023, the Board was strengthened with two NED appointments: Dr Laurence Reilly & Dr Sébastien Goudreau
Incanthera

On 3 June 2024 the Company sold its investment in shares in Incanthera plc. All of the 9,904,319 shares held at the year end were sold at 15p per share realising gross proceeds of £1.4 million. ImmuPharma continues to hold 7,272,740 warrants in Incanthera plc.
Commenting on the statement and outlook Tim McCarthy, CEO and Chairman, said:

"As a Board, we remain focused on the development of our two key late stage clinical assets, P140 (SLE) and P140 (CIDP), and on securing additional partnering deals for each. .We have made significant scientific progress over the last year, including further refinement of the protocol for the P140 (SLE) study and new insights into the MOA of P140, and as a result, we have a high level of confidence of the success of the new study.

We look forward to providing further updates on the progress of this study, together with progress on P140 (CIDP) and our earlier stage programs throughout 2024.

We will also continue to concentrate on further commercial and partnering opportunities. In conjunction with the above objectives, we continue to take prudent measures on managing our cost base.

In closing, we would like to thank our shareholders for their support as well as our staff, corporate and scientific advisers and our partners including CNRS and Avion."