PDC*line Pharma Presents Interim Clinical Results From Last Cohort of Patients in Phase I/II Trial with PDC*lung01 Cancer Vaccine [AACR 2024]

Intermediary results of high dose PDC*lung01 combined with pembrolizumab show mild safety profile, immunological activity and promising tumor response in Non-Small Cell Lung Cancer patients

 

Combination of high dose PDC*lung01 with pembrolizumab in 19 evaluable patients resulted in objective response rate of 63.2% and median progression-free survival of 10.9 months

Liège, Belgium, and Grenoble, France, April 8, 2024 – PDC*line Pharma, a clinical-stage biotech company developing a new class of potent and scalable active immunotherapies for cancers, announces the interim results from the last cohort of patients in its phase I/II clinical trial (PDC-LUNG-101, NCT03970746) with PDC*lung01.

PDC*lung01 is the company’s off-the shelf therapeutic cancer vaccine candidate for Non-Small Cell Lung Cancer (NSCLC). The preliminary data on the last cohort of patients was presented through both an oral presentation and a poster at the American Association for Cancer Research (AACR) Annual Meeting 2024. This data revealed that the high dose PDC*lung01 combined with pembrolizumab shows an immunological activity in a majority of patients and a promising antitumor response in stage IV NSCLC with a mild safety profile.

The phase I/II trial (PDC-LUNG-101) aimed to assess the safety, tolerability, immunogenicity and preliminary clinical activity of PDC*lung01 in NSCLC patients, alone or in combination with anti-PD-1 treatment. PDC*lung01 was administered weekly through both subcutaneous and intravenous routes, in six consecutive doses. The trial was conducted across 17 clinical sites in France, Belgium, Germany, the Netherlands and Poland. PDC*lung01 was administered to a total of 67 evaluable HLA-A*02:01 positive NSCLC patients, at two dose levels and settings:

• As a single agent in the adjuvant setting (cohorts A1: Low Dose, A2: High Dose)
• Combined with standard of care anti-PD-1 monotherapy in first-line stage IV (metastatic) NSCLC patients with a PD-L1 tumor proportion score of ≥50% and no targetable driver mutation (cohorts B1: Low Dose, B2: High Dose)

Clinical activity parameters such as Objective Response Rate (ORR) and Progression-Free Survival (PFS) were assessed only in cohorts B1 and B2. The B2 cohort included 45 patients. PDC*Line is reporting preliminary efficacy results for 19 evaluable patients in the B2 cohort that reached the 9-month PFS mark... PDC*line Pharma's Press Release - 

Sionna Therapeutics Announces Phase 1 Initiation for SION-109 in Cystic Fibrosis

– SION-109 is a small molecule targeting the interface between the ICL4 region and the NBD1 domain of the CFTR protein; designed to complement the company’s NBD1 modulator portfolio –

– First subject dosed in Phase 1 clinical trial –

Boston, MA, January 24, 2024 — Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), announced that the first healthy subject has been dosed in a Phase 1 clinical trial of SION-109 following clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA).

SION-109 is a small molecule designed to target the interface between the intracellular loop 4 (ICL4) region and the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is a complementary mechanism to the company’s portfolio of compounds directly targeting NBD1, including SION-638, the first-in-class NBD1 stabilizer now in Phase 1.

“Our ability to target NBD1 correction is truly novel, and with SION-109 now entering clinical development, we have the opportunity to develop proprietary combination treatments that have the potential to achieve full CFTR correction,” said Mike Cloonan, President and Chief Executive Officer of Sionna. “Advancing our second investigational drug into Phase 1 is an achievement made possible by the tireless work of our research and development teams, who have deep experience in CF and a sharp focus on advancing these programs rapidly to deliver new options for people living with CF and their families.”

CF is caused by genetic mutations in the CFTR protein, an epithelial ion channel that is essential for producing healthy, freely flowing mucus in the airways, digestive system, and other organs. The most common mutation in CFTR, ΔF508, causes NBD1 to unfold at body temperature and severely impairs CFTR function. Complementary targets including ICL4 may enable combination treatments with the potential for full CFTR correction. It is estimated that 90 percent of people living with CF have the genetic mutation ΔF508... Sionna Therapeutics' Press Release - 

NuvoAir’s Air Next Spirometer is FDA-approved for in-home use

The Air Next Spirometer was granted 510k approval for in-home use by the NuvoAir clinical team and its clinical trials partners.

January 25th, 2024 – Boston, MA: NuvoAir Medical announced its innovative Air Next Spirometer has received formal 510(k) clearance for in-home use as a full spirometer. 510(k) approval empowers NuvoAir to scale and expand clinical operations to ensure no patient with heart or lung conditions is left behind.

510(k) clearance allows Air Next to conduct full spirometry at home, providing extremely valuable clinical data that ensures patients can be monitored continuously. High-quality lung data empowers the NuvoAir clinical team to intervene when changes are detected and provide instantaneous care before lung function deteriorates to the point where hospitalizations are necessary. For patients with conditions like COPD, Asthma, or ALS, using this easy-to-use device at home means they don’t have to wait for an in-person appointment or plan the logistics for travel.

“Air Next is capable of providing our care team with high-quality lung measurements from the comfort of our patients’ homes,” says Eric Harker, MD MPH MBA, and Chief Medical Officer at NuvoAir. “Spirometry has traditionally been underutilized as a diagnostic and monitoring tool because of the logistical challenges of getting complex patients to a clinic with regularity. In-home spirometry empowers our practice to provide continuous care to our patients in the home with real-time, high-quality, and actionable clinical data.”

By achieving 510(k) clearance, the Air Next Spirometer can now be used for ATS 2019-compliant spirometry in clinical trials across the US and Europe. “Air Next brings the reliability of in-clinic spirometry right to our patients’ hands,” says Furat Shawki, General Manager of Clinical Trials at NuvoAir. “With in-home approval of ATS 2019-compliant spirometry, we can look forward to scaling our services with our partners across the healthcare ecosystem, including healthcare providers and clinical trials, so we can deliver the cardiopulmonary care that people desperately deserve to the places that need it most.”... NuvoAir Medical's Press Release - 

Thirona : LungQ 3.0.0 receives FDA 510(k) clearance

Nijmegen, the Netherlands (January 22nd, 2024) – Thirona, a global company specializing in advanced analysis of thoracic CT images with artificial intelligence, announced that it has received U.S. Food and Drug Administration (FDA) 510(k) clearance for the latest update of its AI-based clinical software LungQ™ (v3.0.0), making its new features widely available for use in hospitals in the United States.

LungQ 3.0.0 builds on 10 years of clinical trials and 200+ publications to deliver advanced segmentation of pulmonary segments and subsegments.

LungQ 3.0.0 is one of the only FDA-cleared solutions that is capable of using AI to automatically segment the pulmonary segments and subsegments found in the internal anatomy of the lung. Based on this analysis, which includes the identification of structures such as lobes, segments, subsegments, airways, and fissures, the technology performs an analysis of the lung tissue and the fissure completeness, supporting physicians in the diagnosis and documentation of pulmonary tissue images from CT thoracic datasets for each individual patient.

“A clearer understanding of lung anatomy helps enable broader adoption of minimally invasive treatments for lung diseases such as COPD and lung cancer, helping save more healthy lung tissue and lung function capacity. Acting as a map for lung anatomy, LungQ helps guide bronchoscopic navigation, leveraging AI to significantly enhance the precision, accuracy, and efficiency of bronchoscopic and surgical lung interventions,” said Eva van Rikxoort, Founder and CEO of Thirona. “Solutions like LungQ are helping usher in a new era of personalized treatment for lung patients, enabling clinicians all over the world to conduct more advanced, easier-to-perform and less invasive procedures with full confidence.”

The LungQ software first received 510(k) clearance for version 1.1.0 in 2018. With new enhancements, based on deep-learning technology, v.3.0.0 helps guide pulmonologists through the best approach to access various and most peripheral locations within the lungs by delineating pulmonary structures and providing highly accurate reproducible CT values for pulmonary tissue. These insights provide essential, non-invasive quantitative support for diagnosis, treatment planning and follow up examination of lung patients

Rita Priori, CTO at Thirona: “Our AI-based image analysis software LungQ is already used by interventional pulmonologist in Europe and Australia, aiding clinicians through procedures like bronchoscopic lung volume reduction and other interventions. We’re excited to build on the value we’re already seeing in the clinic, helping accelerate innovation in and adoption of a multitude of pulmonary interventions that require high precision on a subsegmental and segmental level, such as lung cancer biopsies, surgical lung volume reduction, lung segmentectomy, ablation procedures, and more.”

The LungQ AI-powered software is approved for clinical use in Europe, the UK and Australia. It is currently used by more than 600 hospitals and has been validated in more than 200 publications globally. The FDA 510(k) cleared LungQ opens tremendous new opportunities for planning and performing localized treatments with maximum precision and accuracy... Thirona's Press Release -

Affimed Provides Clinical Response Update on AFM24-102 Trial in EGFR-wildtype Non-Small Cell Lung Cancer

• Data update from AFM24-102 Phase 1/2a combination study includes 15 heavily pre-treated patients from the EGFR-wildtype (wt) non-small cell lung cancer (NSCLC) expansion cohort
• Follow-up shows three of the initially reported responses have now been confirmed (1 CR, 2 PR) and one still awaiting a confirmatory scan
• Study to be expanded to a total of 40 EGFRwt NSCLC patients
• Enrollment in the EGFR-mutant (mut) NSCLC cohort is ongoing
• Data from both the EGFRwt and EGFRmut cohorts expected in H1 2024

MANNHEIM, Germany, Jan. 08, 2024 — Affimed N.V. (Nasdaq: AFMD) (“Affimed” or the “Company”), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, today provided an update on the clinical response data for the ongoing AFM24-102 phase 1/2a study in EGFRwt NSCLC.

As of January 4, 2024, updated information on the 4 responses in the 15 patients treated in the EGFRwt NSCLC cohort, now show 1 confirmed complete response (CR), 2 confirmed partial responses (PR) and 1 unconfirmed PR awaiting confirmation. Initial data as presented on December 11, 2023, showed 1 confirmed PR as well as 1 unconfirmed CR, and 2 unconfirmed PRs. The patients enrolled in the Phase 1/2a AFM24-102 in the EGFRwt NSCLC cohort of the trial previously had a median of 2 prior lines of therapy, were heavily pretreated, and all patients had progressed on PD-[L]1 targeted therapy.

Based on the promising response data from the EGFRwt NSCLC cohort, Affimed will expand enrollment to 40 patients. Additionally, the Company reported that enrollment of the EGFRmut NSCLC cohort is ongoing. Data from both cohorts are expected in H1 2024.

“We are encouraged by the responses in these patients who had all progressed on PD1 targeting therapy and made the strategic decision to expand this patient cohort,” said Dr. Andreas Harstrick, CMO and interim Chief Executive Officer of Affimed. “There is a significant unmet need for these patients who have exhausted all previous lines of therapy and did not respond to any therapies, including PD-1/PD-L1 treatment. We look forward to sharing more updates in the first half of 2024.”... Affimed's Press Release -

European Medicines Agency Validates Bristol Myers Squibb’s Application for Repotrectinib for the Treatment of Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer and NTRK-Positive Solid Tumors

Application based on data from the registrational TRIDENT-1 and CARE trials showing robust responses and durable activity in these patient populations

If approved, repotrectinib will offer a potential best-in-class treatment for patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer and a new option for patients with NTRK-positive solid tumors, in the European Union

PRINCETON, N.J.- Bristol Myers Squibb (NYSE: BMY) announced that the European Medicines Agency (EMA) has validated its marketing authorization application for the next-generation tyrosine kinase inhibitor (TKI) repotrectinib as a treatment for ROS1 TKI-naïve and -pretreated adult patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) and TKI-naïve and -pretreated adult and pediatric patients 12 years and older with NTRK-positive locally advanced or metastatic solid tumors. The application was based on results from the registrational Phase 1/2 TRIDENT-1 trial (adult patients with ROS1-positiveNSCLCor NTRK-positivesolid tumors) and CARE study (pediatric patients with NTRK-positivesolid tumors). Validation of the application confirms the submission is complete and begins the EMA’s centralized review procedure.

“While there are existing TKIs for the treatment of ROS1-positive non-small cell lung cancer and NTRK-positive solid tumors, there is still an undeniable need for new options that may offer patients more durable responses, including those with metastatic disease to the brain, and may address the issue of treatment resistance for patients with these aggressive and rare tumors,” said Joseph Fiore, executive director, global program lead, repotrectinib, Bristol Myers Squibb. “The clinical trial results with repotrectinib highlight the potential for this next-generation therapy to become a best-in-class option for patients with tumors harboring ROS1 fusions and a new option for those with NTRK-fusion positive tumors, two groups who each continue to face a high unmet medical need. Today’s validation by the European Medicines Agency brings us another step closer to our goal of making this new targeted therapy available to patients in Europe.”

In the TRIDENT-1 and CARE trials, repotrectinib demonstrated clinically meaningful response rates in patients across ROS1-positive NSCLC and NTRK-positive solid tumor cohorts. Durability of response was robust and intracranial responses were observed in both settings, as well as in patients whose tumors harbor common resistance mutations. The safety profile of repotrectinib was well characterized and generally manageable with routine standard-of-care treatments. The study remains ongoing to assess long-term outcomes and additional endpoints across patient populations with ROS1-positive locally advanced or metastatic NSCLC and NTRK-positive locally advanced or metastatic solid tumors.

Additionally, in November 2023 the U.S. Food and Drug Administration approved Augtyro™ (repotrectinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive NSCLC.

Bristol Myers Squibb thanks the patients and investigators involved with the TRIDENT-1 and CARE clinical trials.

Turning Point Therapeutics is a wholly owned subsidiary of the Bristol-Myers Squibb Company. As of August 2022, Bristol Myers Squibb acquired the company, including its asset repotrectinib... Bristol Myers Squibb’s Press Release -