On November 13, 2024 Pulmatrix ("Pulmatrix") (Nasdaq: PULM), a clinical-stage biopharmaceutical company, reported a merger agreement with Cullgen Inc. ("Cullgen"), a privately-held, clinical-stage biopharmaceutical company applying its proprietary targeted protein degradation uSMITE platform to discover and advance therapeutics for the treatment of cancer and other diseases (Press release, Pulmatrix, NOV 13, 2024, View Source [SID1234648284]). Cullgen utilizes its proprietary technology platform, uSMITE, featuring novel E3 ligands, to build the next generation of targeted protein degraders and degrader-antibody conjugates ("DACs").

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In 2023 Cullgen completed a Series C financing led by AstraZeneca-CICC Venture Capital Partnership, and also announced a strategic partnership with Astellas Pharma Inc.

About the Proposed Transaction

Subject to the terms and conditions of the merger agreement, and upon the closing of the merger, pre-merger Pulmatrix stockholders are expected to own approximately 3.6% of the combined company, and pre-merger Cullgen stockholders are expected to own approximately 96.4% of the combined company which will operate under the name Cullgen Inc., be headquartered in San Diego, CA and trade on The Nasdaq Capital Market (Nasdaq). Pulmatrix stockholders will also receive a special cash dividend to the extent that Pulmatrix’s net cash at closing exceeds $2.5 million, subject to certain adjustments.

The transaction is expected to close by the end of March 2025, subject to obtaining stockholder and CSRC approval.

Management and Board of Directors

At the effective time of the merger, the executive officers of the combined company will be led by Ying Luo, Ph.D. Cullgen’s Chairman and CEO. The Cullgen Board of Directors will be supplemented by one representative of Pulmatrix.

Ying Luo, Ph.D., commented, "I’m delighted to announce this planned merger with Pulmatrix, which comes at a pivotal moment in the evolution of our company as we advance our pipeline of targeted protein degraders into clinical development for cancer and other diseases. Listing on Nasdaq will help fuel our growth and enable us to unlock the full potential of our technology platform, including our plans to develop and advance degrader-antibody conjugates and additional targeted protein degraders into the clinic."

Peter Ludlum, Interim Chief Executive Officer of Pulmatrix, added, "Following a thorough evaluation of strategic alternatives, the Pulmatrix board of directors and management team believe that this anticipated transaction represents an opportunity to deliver value to our stockholders. We anticipate that this merger will allow our stockholders to participate in Cullgen’s promising research and development activities and also provide a benefit in the form of a potential dividend component immediately prior to the close of the merger. Prior to transaction closing, we will attempt to increase cash available for the special dividend by divesting corporate assets including PUR1800, PUR3100, and the patent portfolio for iSPERSE."

Cullgen Pipeline Overview and Development Milestones

Cullgen currently has three degrader programs that are in or about to initiate Phase 1 clinical testing. CG001419 is a first-in-class, selective, clinically active, oral pan-TRK degrader that is being studied in two separate clinical trials—one for solid tumors, and the other for the treatment of acute and chronic pain. With respect to the cancer trial, Cullgen has dosed ten patients thus far with no observed dose-limiting toxicity, treatment-related serious adverse events or grade ≥ 3 treatment related adverse events. Regarding the pain trial, Cullgen recently received HREC (Australia) approval to begin enrolling patients to evaluate the safety and pharmacokinetic characteristics of CG001419 in healthy volunteers. Cullgen anticipates enrolling the first patient in early 2025. This program aims to provide a new non-opioid non-NSAID analgesic which can fulfill unmet medical needs in the field of pain.

Cullgen is also evaluating CG009301, a GSPT1 degrader for the treatment of blood cancers including relapsed/refractory acute myeloid leukemia (AML), higher-risk myelodysplastic syndrome (HR-MDS), and acute lymphoblastic leukemia (ALL). Cullgen has received IND allowance from the China CDE for this product candidate and Cullgen anticipates dosing the first patient in the first quarter of 2025. CG009301 also has the potential to be used for the treatment of solid tumors harboring MYC amplification.

In addition to these three clinical programs, Cullgen is also advancing several other targeted protein degraders and DACs, predominantly for the treatment of cancer and autoimmune disease, including a program targeting a cell cycle protein that has been partnered with Astellas Pharma Inc.

Webcast Information

Management will host an informational webcast that will be made available to access at 12:00 pm ET today. A link to the webcast can be found in the Investor Events and Presentations section of the Pulmatrix website at and in the News & Events section of Cullgen’s website at .

On November 13, 2024 Servier reported that it will present data at the Annual Meeting for the American Society of Hematology (ASH) (Free ASH Whitepaper), December 7-10, 2024 in San Diego, California. Data presentations will focus on the company’s clinical and preclinical studies in IDH-mutated acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), providing new insights on existing programs and a better understanding of the challenges faced by people living with hematological malignancies (Press release, Servier, NOV 13, 2024, View Source [SID1234648308]).

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"Servier is driven to deliver innovative therapies for diseases where patient needs are not yet sufficiently met and where we believe we can have the greatest positive impact," said Becky Martin, PhD, Chief of Medical at Servier Pharmaceuticals. "We look forward to sharing data at this year’s ASH (Free ASH Whitepaper) annual meeting that build upon our expertise in developing medicines for patients living with hematological malignancies, including IDH-mutated AML and MDS."

On November 13, 2024 Curium, a world leader in nuclear medicine, reported that its ECLIPSE trial met its primary endpoint (Press release, Curium Pharma, NOV 13, 2024, View Source [SID1234648264]). ECLIPSE is a pivotal Phase 3, multi-center, open-label, randomized clinical trial comparing the safety and efficacy of 177Lu-PSMA-I&T (INN: lutetium (177Lu) zadavotide guraxetan) versus hormone therapy in patients with metastatic castration-resistant prostate cancer.

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The ECLIPSE trial demonstrated a statistically significant and clinically meaningful improvement in the median radiographic progression-free survival (rPFS) of patients with prostate-specific membrane antigen (PSMA) positive metastatic castration-resistant prostate cancer (mCRPC) after treatment with up to 6 doses of 200 mCi (7.4 GBq) of 177Lu-PSMA-I&T in patients previously treated with an androgen receptor pathway inhibitor (ARPI) compared to a change in ARPI.

Sakir Mutevelic, MD, Curium’s Chief Medical Officer said: "This is a significant accomplishment for Curium, demonstrating in the pivotal confirmatory ECLIPSE trial a statistically significant and clinically meaningful benefit of PSMA-targeted radioligand therapy with 177Lu-PSMA-I&T for patients with mCRPC. ECLIPSE is the first Phase 3 trial investigating a 200 mCi (7.4 GBq) dose of 177Lu-PSMA-I&T administered every six weeks for up to six doses, demonstrating clinical benefit, in mCRPC patients before receiving taxane-based chemotherapy. Curium will continue to work with the FDA as the clinical trial data matures, on a regulatory submission plan for this potentially important product for patients, their caregivers, and the healthcare providers treating prostate cancer."

Michael Patterson, CEO, Curium North America added: "The ECLIPSE achievement of its primary endpoint represents an important clinical milestone in the development of our prostate theranostic program. This underscores Curium’s continued commitment and focus on nuclear medicine diagnostics and therapeutics. Further, the announcement of the opening of Curium’s Netherlands facility for the production of 177Lutetium in September 2024, bolsters Curium’s supply chain and ensures manufacturing reliability. Curium will continue to work to fulfill its mission of redefining the experience of cancer through our trusted legacy in nuclear medicine by ensuring unrestricted access to this important product, if approved."

On November 13, 2024 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, reported an update on the company’s development pipeline and announced financial results for the third quarter ended September 30, 2024 (Press release, Quince Therapeutics, NOV 13, 2024, View Source [SID1234648285]).

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Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, "We are pleased to report accelerating enrollment of our pivotal Phase 3 NEAT clinical trial in Ataxia-Telangiectasia (A-T). As of today, we have enrolled 32 patients with A-T across clinical sites in the U.S., U.K., and European Union. Additionally, the majority of planned NEAT study sites are now activated and open for enrollment. We expect enrollment momentum to continue as we work toward our commitment to complete enrollment in the second quarter of 2025 and report topline results in the fourth quarter of 2025."

Pivotal Phase 3 NEAT Clinical Trial

Enrolled 32 participants to date in the company’s Phase 3 NEAT (Neurologic Effects of EryDex on Subjects with A-T; IEDAT-04-2022/NCT06193200) clinical trial to evaluate the neurological effects of EryDex in patients with A-T.
Quince plans to enroll approximately 86 patients with A-T ages six to nine years old (primary analysis population) and approximately 20 patients with A-T ages 10 years or older.
Participants who complete the full treatment period, complete study assessments, and provide informed consent will be eligible to transition to an open label extension study, which will begin in the fourth quarter of 2024.
Pivotal Phase 3 NEAT clinical trial is being conducted under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration (FDA).
Expect to report Phase 3 NEAT topline results in the fourth quarter of 2025 with a New Drug Application (NDA) submission to the FDA and a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in 2026, assuming positive study results.
NEAT is an international, multi-center, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of the company’s lead asset, EryDex (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells), in patients with A-T.
Participants will be randomized (1:1) between EryDex or placebo and treatment will consist of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last visit completion in a rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo as per the SPA agreement with the FDA.
Pipeline and Corporate Updates

Participation at scientific congresses, including a poster presentation of safety data from the previously completed ATTeST study at the 53rd Child Neurology Society Annual Meeting. Quince is also sponsoring and participating at the 2024 International Congress for Ataxia Research in November 2024 with two poster presentations, including growth and bone mineral density in patients with A-T treated with EryDex, and an analysis of the International Cooperative Ataxia Rating Scale (ICARS) subcomponent scores in patients with A-T.
Generating Phase 2 clinical trial study designed to evaluate EryDex for the potential treatment of patients with Duchenne muscular dystrophy (DMD), including those with corticosteroid intolerance, who represent the majority of the DMD population. Quince plans to initiate a DMD Phase 2 study in 2025, which the company expects to conduct utilizing capital efficient study approaches.
Completed evaluation process of other potential rare disease indications beyond A-T and Duchenne muscular dystrophy for EryDex where chronic corticosteroid treatment is – or has the potential to become – a standard of care, if there were not corticosteroid-related safety concerns. The prioritized list of other potential rare disease targets under consideration includes: 1) autoimmune hepatitis, 2) dermatomyositis, 3) pemphigus vulgaris, 4) Hashimoto’s encephalopathy, 5) Becker muscular dystrophy, 6) pediatric lupus, 7) juvenile idiopathic arthritis, 8) myasthenia gravis, 9) limb-girdle muscular dystrophy, 10) chronic inflammatory demyelinating polyradiculoneuropathy, and 11) pulmonary sarcoidosis.
Third Quarter and Year-to-Date 2024 Financial Results

Reported cash, cash equivalents, and short-term investments of $47.8 million for the third quarter ended September 30, 2024. Quince expects its existing cash runway to be sufficient to fund the company’s capital efficient development plan through Phase 3 NEAT topline results and into 2026.
Expect strong cash position to fully fund lead asset, EryDex, through Phase 3 NEAT topline results in the fourth quarter of 2025 and prepare for NDA and MAA submissions in 2026, assuming positive study results. This includes approximately $20 million for the NEAT clinical trial and approximately $15 million in direct trial costs for an open label extension study.
Reported research and development (R&D) expenses of $4.9 million for the third quarter ended September 30, 2024. R&D expenses during the quarter primarily included costs related to ongoing Phase 3 NEAT clinical trial activities and related manufacturing costs.
Reported general and administrative (G&A) expenses of $3.6 million for the third quarter ended September 30, 2024. G&A expenses for the quarter primarily included personnel-related and stock-based compensation expenses, commercial planning and new product planning expenses, and other professional administrative costs.
Reported a net loss of $5.5 million, or a net loss of $0.13 per basic and diluted share, for the third quarter ended September 30, 2024. Weighted average shares outstanding for the quarter were 43.2 million.
Reported net cash used in operating activities of $24.4 million for the nine months ended September 30, 2024, which included a net loss of $44.4 million for the period, adjusted for $22.4 million of non-cash items, including $17.1 million goodwill impairment charge, $2.1 million change in the fair value of contingent consideration liabilities, $3.6 million in stock-based compensation, a net increase in operating assets of $2.5 million, and a net increase in accounts payable, accrued expenses, and other current liabilities of $0.1 million. Additionally, Quince made a cash milestone payment of $5 million to EryDel shareholders in the third quarter of 2024 following the achievement of the first patient enrolled in the NEAT study in the second quarter of 2024.

On November 13, 2024 Continuity Biosciences, LLC, a new bioscience company dedicated to developing and commercializing cutting-edge technologies for cell reprogramming, immune modulation, and drug delivery, reported its official launch (Press release, Continuity Biosciences, NOV 13, 2024, View Source [SID1234648309]). The company seeks to bridge biopharmaceutical and medical technologies, establishing a new standard for patient-focused treatments for chronic and complex diseases.

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Founded by a team of experienced scientists and industry leaders, Continuity Biosciences aims to enhance emerging therapies, such as cell therapy, by fully leveraging delivery technology. The team includes Bob Whitehead (Executive Chairman), a veteran in the bioscience field known for successful strategic exits and initiatives; Ramakrishna Venugopalan, PhD (Co-Founder & CEO), a former senior executive at AbbVie with expertise in drug delivery for products like Skyrizi and Vyalev; and Alessandro Grattoni, PhD (Chief Scientific Advisor), Chair and Professor of the Department of Nanomedicine at Houston Methodist Hospital and a leader in advanced drug delivery.

"We are thrilled to have licensed several technology platforms from Houston Methodist Hospital and to introduce Continuity Biosciences to the biotechnology and investment communities," said Ramakrishna Venugopalan, Co-Founder & CEO. "There is immense potential at the intersection of biopharmaceuticals and medical technologies, especially in creating combination products that enhance novel therapies. We plan to expand our technology portfolio to provide our partners with diverse strategies for delivering their treatments."

Dr. Grattoni’s pioneering research, along with Continuity’s licensed technologies, focuses on developing implantable nanofluidic systems. These technologies facilitate cell reprogramming, molecular sieving, immune modulation, and sustained therapeutic release over ultra-long periods. Supported by significant funding from organizations like the National Institute of Health, Department of Defense, United States Agency for International Development, Juvenile Diabetes Research Foundation (now Breakthrough T1D), ISS National Lab, and key pharmaceutical partners and foundations, these platforms have vast potential for preventing and treating chronic conditions including cancer, autoimmune, metabolic and infectious diseases. They also offer versatile applications for long-acting drug delivery and in vivo cell reprogramming systems suited for advanced cell and gene therapies.