On December 30, 2024 Immedica Pharma AB (Immedica), a leading global rare disease company, and Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company committed to improving the lives of patients with seizure disorders, reported that they have entered into an agreement and plan of merger under which Immedica has agreed to acquire Marinus, by means of a tender offer and subsequent merger (Press release, Immedica Pharma, DEC 30, 2024, View Source [SID1234649366]).

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The acquisition complements and further strengthens Immedica’s global rare disease business by adding ZTALMY (ganaxalone) oral suspension, CV, a neuroactive steroid gamma-aminobutyric acid (GABA)-A receptor positive modulator, approved by the U.S. Food and Drug Administration (FDA) in March 2022 for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two years of age and older.

Transaction rationale and details in brief:

Adds global rights to ZTALMY, a commercial-stage rare neurology medicine approved by FDA, the European Commission (EC), the UK Medicines and Healthcare products Regulatory Agency (MHRA) and the National Medicines Product Administration (NMPA) in China with potential for further approvals worldwide.
Accelerates Immedica’s growth into the North American market, providing an immediate revenue-generating rare disease product and an experienced commercial team upon closing of the transaction.
Acquisition is expected to accelerate Immedica’s revenue growth, adding a commercial-stage asset in the United States, with the potential for further expansion globally.
Immedica to commence a cash tender offer to acquire all issued and outstanding shares of Marinus for USD 0.55 per share, corresponding to an implied enterprise value of approximately USD 151 Million.
Transaction is expected to close in Q1 2025.
"The acquisition of Marinus represents a transformative step in Immedica’s journey to further strengthen our position as a leading rare disease company. By adding ZTALMY to our portfolio, we significantly strengthen our capabilities and expand our presence in the United States, marking a new chapter in our mission to deliver impactful therapies for underserved patient populations," said Anders Edvell, M.D. Ph.D. and Chief Executive Officer of Immedica.

"Immedica is dedicated to addressing significant unmet medical needs in rare diseases, ensuring patients receive the innovative treatments they deserve. Within CDD, patients with refractory seizures face particularly challenging circumstances due to insufficiently effective existing therapies. The addition of ZTALMY allows us to offer a differentiated solution, with the potential to improve care and outcomes for these patients," he concluded.

"I am proud of the dedication and passion of our team at Marinus, which allowed us to deliver the first and only FDA-approved treatment for seizures associated with CDKL5 deficiency disorder in patients two years of age and older," said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. "With a shared commitment to improving the lives of rare disease patients, this acquisition is expected to enable ZTALMY to make an even greater impact on patients, while providing meaningful value for Marinus’ stockholders."

Transaction details

Under the terms of the merger agreement, Immedica, through a wholly owned, direct subsidiary, will initiate a tender offer to acquire all the outstanding shares of Marinus common stock for a cash purchase price of USD 0.55 per share, representing a premium of 48% based on Marinus’ closing share price as of December 27th and a premium of 97%, based on the 30-day volume-weighted average price of USD 0.28 per share preceding the announcement of the transaction. The Board of Directors of Marinus has unanimously approved the transaction and recommended that the stockholders of Marinus tender their shares in the tender offer. Immedica has received an undertaking from each director and named executive officer of Marinus to tender their respective shares in favor of the transaction.

The transaction represents the culmination of Marinus’ review of strategic alternatives, which it announced on October 24, 2024, with the goal of maximizing value for its stockholders.

The closing of the tender offer will be subject to customary conditions, including the tender of shares which represent at least a majority of the total number of Marinus’ outstanding shares of common stock. Upon the successful completion of the tender offer, Immedica would acquire any shares of Marinus’ common stock not tendered through a second-step merger effected for the same per share consideration. The transaction is expected to close in Q1 2025.

Advisors

MTS Health Partners LP is acting as Immedica’s exclusive financial advisor in connection with the transaction. Gibson, Dunn & Crutcher LLP is acting as legal counsel to Immedica and Fuchs Patentanwälte Partnerschaft mbB is acting as intellectual property counsel on this transaction, Barclays Capital Inc. is acting as Marinus´ exclusive financial advisor in connection with the transaction. Hogan Lovells LLP is acting as legal counsel to Marinus on this transaction.

About ZTALMY (ganaxolone) oral suspension

ZTALMY (ganaxolone) is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects. It is a prescription medicine that has been approved by the U.S. FDA, EC, the MHRA, and the China NMPA for appropriate patients with CDKL5 deficiency disorder.

U.S. Prescribing Information for ZTALMY (ganaxolone) oral suspension CV.

European Union Summary of Product Characteristics for ZTALMY.

On December 30, 2024 RenovoRx, Inc. ("RenovoRx" or the "Company") (Nasdaq: RNXT), a life sciences company developing novel targeted oncology therapies and commercializing RenovoCath, a novel, FDA-cleared delivery system, reported its strong intellectual property (IP) position as it moves forward with commercial plans and clinical trial advancement in 2025, utilizing its novel Trans-Arterial Micro-Perfusion (TAMP) therapy platform (Press release, Renovorx, DEC 30, 2024, View Source [SID1234649365]).

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Earlier this year, RenovoRx published a new international patent application for its TAMP therapy platform to expand its IP portfolio. RenovoRx already holds strong IP including coverage of its proprietary TAMP therapy platform and RenovoCath delivery system, with 8 issued and 6 pending U.S. patents, and 10 issued and 7 pending patents outside of the US, including its international application published in 2024.

The new published international patent application WO2024102497 describes methods and tools for delivering treatments (including antibodies, nanoparticles, or charged molecules) directly to specific tissues. This is accomplished using micro-vessels (called the vasa vasorum) that supply the outer walls of larger blood vessels near the specific target tissue to be treated. This invention opens innovative and effective ways to target and deliver cancer treatments in local tissue beyond the current standard-of-care, systemic (intravenous "IV") treatment.

"Delivering therapeutics through micro-vessels around the artery via our novel Trans-Arterial Micro-Perfusion technique is central to our treatment paradigm and value proposition," said Ramtin Agah, MD, Chief Medical Officer and Founder of RenovoRx. "We already hold strong IP protection for our technology, and our 2024 pending patent further describes and seeks protection for the novelty of our approach."

TAMP is designed to ensure precise therapeutic delivery across the arterial wall near the tumor site to bathe the target tumor, while potentially minimizing a therapy’s toxicities versus systemic intravenous therapy. RenovoRx’s novel approach to targeted treatment offers the potential for increased safety, tolerance, and improved efficacy.

"As we move forward in 2025 with our commercial plans for RenovoCath and the advancement of our pivotal Phase III TIGeR-PaC clinical trial, both of which are driven by TAMP, it is important to highlight the strong IP foundation we have and continue to expand. The new patent application filed earlier this year would further the value that TAMP can offer the oncology community as an innovative and effective approach to drug delivery," said Shaun Bagai, CEO, RenovoRx. "Once approved, it will incrementally extend our IP coverage and increase the commercial value proposition for both TAMP and RenovoCath."

RenovoRx’s pivotal ongoing Phase III TIGeR-PaC clinical trial is evaluating its first product candidate, a novel investigational oncology drug-device combination utilizing the FDA-cleared RenovoCath device via TAMP for the intra-arterial administration of the chemotherapy gemcitabine. RenovoRx currently anticipates completion of both patient enrollment and the second interim analysis for TIGeR-PaC by the end of the first half of 2025.

Moreover, RenovoRx recently announced the receipt of its first purchase orders for RenovoCath devices. This milestone marks a positive continuation of RenovoRx’s previously announced efforts to commercialize RenovoCath as a standalone device to be used by doctors in accordance with its FDA-cleared instructions for use. RenovoCath is powered by TAMP.

About RenovoCath

Based on its FDA clearance, RenovoCath is intended for the isolation of blood flow and delivery of fluids, including diagnostic and/or therapeutic agents, to selected sites in the peripheral vascular system. RenovoCath is also indicated for temporary vessel occlusion in applications including arteriography, preoperative occlusion, and chemotherapeutic drug infusion. For further information regarding our RenovoCath Instructions for Use ("IFU"), please see: IFU-10004-Rev.-F-Universal-IFU.pdf.

About the TIGeR-PaC Clinical Trial

TIGeR-PaC is an ongoing Phase III randomized multi-center study evaluating the proprietary TAMP (Trans-Arterial Micro-Perfusion) therapy platform for the treatment of Locally Advanced Pancreatic Cancer (LAPC.) RenovoRx’s first product candidate using the TAMP technology, is a novel investigational oncology drug-device combination utilizing the Company’s FDA-cleared RenovoCath device for the intra-arterial administration of chemotherapy, gemcitabine.

The first interim analysis in the Phase III clinical trial was completed in March 2023, with the Data Monitoring Committee recommending a continuation of the study. The study’s primary endpoint is an Overall Survival benefit with secondary endpoints including reduced side effects versus standard of care. The second interim analysis for this study will be triggered by the 52nd event (i.e., patient death), which is estimated to occur in late 2024 or early 2025. The second interim data readout would follow thereafter, with the timing for such readout depending on customary factors such as time needed for analysis. RenovoRx is also aiming to complete patient enrollment in the TIGeR-PaC study in the first half of 2025.

On December 30, 2024 Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) reported that Bristol Myers Squibb received U.S. Food and Drug Administration (FDA) approval for Opdivo Qvantig (nivolumab and hyaluronidase-nvhy) co-formulated with Halozyme’s ENHANZE drug delivery technology for subcutaneous use in most previously approved adult, solid tumor intravenous (IV) Opdivo indications as monotherapy, monotherapy maintenance following completion of Opdivo plus Yervoy (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib (Press release, Halozyme, DEC 30, 2024, View Source [SID1234649364]). Opdivo Qvantig is the first and only subcutaneously administered PD-1 inhibitor.

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The subcutaneous administration of Opdivo Qvantig is faster, with a three- to five-minute administration time compared to 30 minutes for IV Opdivo.* Subcutaneous administration may offer flexibility to receive treatment where it is best for patients and their providers, may reduce steps required for preparation and time needed for administration.

"We are pleased Opdivo Qvantig, which is co-formulated with our ENHANZE drug delivery technology, is now FDA-approved as the first and only subcutaneously administered PD-1 inhibitor in the U.S.," said Dr. Helen Torley, president and chief executive officer of Halozyme. "This approval represents our ninth co-formulated product and is yet another example of how Halozyme’s innovative ENHANZE technology is enabling greater flexibility and optionality for patients."

The FDA approval is based on the results from the Phase 3 randomized, open-label CheckMate-67T trial, which was a noninferiority trial evaluating Opdivo Qvantig co-formulated with Halozyme’s proprietary recombinant human hyaluronidase (rHuPH20), compared to intravenous Opdivo, in adult patients with advanced or metastatic clear cell renal cell carcinoma who received prior systemic therapy. In the trial, noninferiority was demonstrated for the co-primary endpoints of time-averaged concentration over 28 days (Cavgd28) and minimum concentration at steady state (Cminss) of Opdivo Qvantig vs. IV Opdivo. The geometric mean ratio (GMR) for Cavgd28 was 2.10 (90% CI: 2.00-2.20) and the GMR for Cminss was 1.77 (90% CI: 1.63-1.93). As a key powered secondary endpoint, the overall response rate (ORR) in the Opdivo Qvantig arm (n=248) was 24% (95% CI: 19-30) compared with 18% (95% CI: 14-24) in the IV Opdivo arm (n=247) showing that Opdivo Qvantig has similar efficacy compared to IV Opdivo as assessed by Blinded Independent Central Review (BICR).

Select Safety Profile from CheckMate-67T

Serious adverse reactions occurred in 28% of patients receiving Opdivo Qvantig. The most frequent serious adverse reactions reported in >1% of patients who received Opdivo Qvantig were pleural effusion (1.6%), pneumonitis (1.6%), hyperglycemia (1.2%), hyperkalemia (1.2%), hemorrhage (1.2%) and diarrhea (1.2%). The most common adverse reactions (reported in ≥10% of patients) were fatigue (20%), musculoskeletal pain (31%), pruritus (16%), rash (15%), arthralgia (12%) and cough (11%). Fatal adverse reactions occurred in 3 (1.2%) patients who received Opdivo Qvantig; these included myocarditis, myositis, and colitis complications. Study therapy was discontinued in 10% of patients due to adverse reactions. The safety profile of Opdivo Qvantig was comparable with the safety profile of IV Opdivo.

On December 30, 2024 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported the completion of enrollment in LOTIS-5, the Phase 3 confirmatory trial evaluating ZYNLONTA (loncastuximab tesirine-lpyl) in combination with rituximab (Lonca-R) in patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) (Press release, ADC Therapeutics, DEC 30, 2024, View Source [SID1234649363]). ZYNLONTA previously received accelerated approval for the treatment of r/r DLBCL after two or more lines of systemic therapy from the FDA in 2021.

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"This milestone for LOTIS-5 brings us a step closer to providing a potential combination treatment in the 2L+ DLBCL setting that we believe will offer competitive efficacy with favorable safety and a convenient dosing schedule, well-suited for use in patients across care settings," said Mohamed Zaki, MD, PhD, Chief Medical Officer of ADC Therapeutics. "We anticipate sharing topline results of the primary endpoint analysis by the end of 2025 once the pre-specified number of events is reached and potentially submitting our supplemental BLA to the U.S. Food and Drug Administration (FDA) in the first quarter of 2026."

The randomized, open‐label, two‐part, two‐arm, multicenter study is designed to confirm accelerated approval and may support potential label expansion into 2L+ in combination with rituximab. Twenty patients were enrolled in part 1 of a non-randomized safety run‐in. As previously reported, the results showed an overall response rate (ORR) by central review of 80% (16/20) with a complete response (CR) rate of 50% (10/20) and no new safety signals.

In part 2, patients with 2L+ DLBCL are randomized 1:1 to receive fixed-dose ZYNLONTA with rituximab or rituximab‐gemcitabine‐oxaliplatin (R‐GemOx). The primary endpoint of LOTIS-5 is progression-free survival with secondary endpoints of overall survival, ORR, CR rate and duration of response as well as frequency and severity of adverse events. Topline results of the primary endpoint analysis are anticipated by the end of 2025 once the required number of pre-specified events is reached followed by regulatory submission to the FDA in Q1 2026 and potential approval in late 2026.

For more information about LOTIS-5, please visit View Source (identifier NCT04384484).

About ZYNLONTA (loncastuximab tesirine-lpyl)
ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at www.ZYNLONTA.com.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On December 30, 2024 Bridge Biotherapeutics (KQ288330), a clinical-stage biotech company based in South Korea developing novel drugs for fibrosis and cancer, reported that James Jungkue Lee, Founder and CEO of Bridge Biotherapeutics, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2025 (Press release, Bridge Biotherapeutics, DEC 30, 2024, View Source [SID1234649362]).

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Location: Georgian Room (Mezzanine Level) at The Westin St. Francis

Date: Thursday, January 16, 2025

Time: 9:45-10:25 a.m. PST

During the conference, Bridge Biotherapeutics will also meet with potential partners and investors as the Company is looking for Business Development opportunities for further development of BBT-877, a novel drug candidate for idiopathic pulmonary fibrosis (IPF). Top-line results from the Phase 2 study of BBT-877 are expected in April 2025.