Liminal BioSciences Gets FDA OK for License Application for Ryplazim

  Liminal BioSciences Inc. (Nasdaq: LMNL) ("Liminal BioSciences" or the "Company") announced today that the U.S. Food & Drug Administration (FDA) has approved Ryplazim® (plasminogen, human-tvmh) ("Ryplazim®") for the treatment of patients with plasminogen deficiency type 1 (hypoplasminogenia) through its subsidiary, Prometic Biotherapeutics Inc., holder of the biological license application ("BLA") for Ryplazim®. With this approval, Ryplazim® becomes the first FDA approved therapy for this rare genetic disorder.

The efficacy of Ryplazim® in pediatric and adult patients with plasminogen deficiency type 1 was evaluated in a single-arm, open-label clinical trial. A total of 15 patients who had a baseline plasminogen activity level between <5% and 45% of normal were enrolled. All patients received Ryplazim® at a dose of 6.6 mg/kg administered every two to four days for 48 weeks to achieve at least an increase of individual trough plasminogen activity by an absolute 10% above baseline and to treat the clinical manifestations of the disease. Ryplazim® was well tolerated in the clinical study (See Important Safety Information).

Efficacy was established on the basis of overall rate of clinical success at 48 weeks defined as 50% of patients with visible or other measurable non-visible lesions achieving at least 50% improvement in lesion number/size, or functionality impact from baseline. All patients with any lesion at baseline showed at least 50% improvement in the number or size of their lesions.

"Receiving our first drug approval is a major milestone for Liminal and for the patients, caregivers, and physicians who have been with us every step of the way in this important research effort. We are very pleased that Ryplazim® will be available to US patients suffering from congenital plasminogen deficiency," said Bruce Pritchard, the CEO of Liminal BioSciences. "The receipt of a Rare Pediatric Disease Priority Review Voucher (PRV) also has the potential to provide Liminal with non-dilutive cash to support our ongoing efforts to advance and expand our small molecule R&D strategy".

https://finance.yahoo.com/news/liminal-biosciences-announces-fda-approval-193000996.html

AstraZeneca IMFINZI Shows Unprecedented Survival in Unresectable Stage III Lung Cancer

 

With 43% Of Patients Surviving Five Years

Long-term data from PACIFIC Phase III trial at ASCO showed 33% of patients remained progression-free at five years

Longest-ever survival reported in a Phase III immunotherapy trial in this setting

https://finance.yahoo.com/news/imfinzi-demonstrated-unprecedented-survival-unresectable-110000358.html

Illumina: GRAIL Presents Interventional PATHFINDER Study Data at 2021 ASCO

 

Introduces Galleri, a Groundbreaking Multi-Cancer Early Detection Blood Test

Galleri’s ability to detect more than 50 types of cancer with a single blood draw could transform early cancer detection as a complement to existing screenings; test now available in U.S. by prescription only

GRAIL, Inc., a healthcare company whose mission is to detect cancer early, today presented the first results from the interventional PATHFINDER study evaluating Galleri™, a multi-cancer early detection (MCED) blood test. The results, presented at the 2021 ASCO Annual Meeting, support Galleri’s performance in clinical settings. The company also announced today that Galleri is now available in the U.S. by prescription only.

"The interim results of PATHFINDER demonstrate that a routine blood test is capable of detecting many different cancers even before symptoms arise, an approach that has significant potential advantages," said Dr. Tomasz M. Beer, deputy director at the OHSU Knight Cancer Institute and presenting author. "Most importantly, it can detect cancers that have no recommended screening tests today, and more than two-thirds of cancers go unscreened for this reason. These results are a pivotal step toward extending early detection to many more types of cancer."

https://www.yahoo.com/now/grail-presents-interventional-pathfinder-study-110000124.html

Protalix Shares Hits 52-Week Low After Adverse Event Reported In Fabry Disease Trial

 

• Protalix BioTherapeutics Inc (NYSE: PLX) and Chiesi Farmaceutici S.p.A. have provided an update on the clinical development of pegunigalsidase alfa (PRX–102) for the proposed treatment of Fabry disease.

• PRX–102 is currently being studied in Phase 3 BALANCE trial evaluating the safety and efficacy of 1 mg/kg of PRX–102 dosed every two weeks compared to agalsidase beta (Fabrazyme).

• The primary endpoint of the study is the comparison of mean annualized changes (slope) of the eGFR (CKD-EPI) after completion of at least 12 months of treatment between the two treatment arms.

• The initial top-line results show that the lower boundary of the confidence interval for the mean difference between the two treatments was below the non-inferiority margin pre-specified in the Intention to Treat analysis set and above such limit in the Intention to Treat analysis set.

• At the time of this analysis (n=77), two patients discontinued participation due to treatment-emergent adverse events - one discontinued participation due to a related adverse event. No deaths were registered.

• Unblinded final data are anticipated in Q2 of 2022 after all remaining patients have completed the 24-month treatment period.

• Despite reporting safety issues, the company will move forward with marketing application in Europe PRX–102 in Fabry disease.

• Regarding the regulatory process in the U.S., the companies plan to submit a Type–A meeting request with the FDA to discuss the path for approval of PRX–102.

• In April, the companies received FDA Complete Response Letter for PRX‑102 for Fabry disease, though no safety concerns were noted in the letter.

https://finance.yahoo.com/news/protalix-shares-hits-52-week-161025988.html

#ASCO21: Merus Presents Clinical Data on Zenocutuzumab in NRG1-fusion Cancers

 61 patients with NRG1+ cancer have been enrolled, including 45 patients evaluable for response as of the April 13, 2021 data cutoff date

- Encouraging early clinical activity observed, with confirmed responses in 5 of 12 patients with pancreatic cancer (42%) and in 13 of 45 patients across several NRG1+ tumor types (29%)
- Zenocutuzumab continues to be well tolerated with a favorable safety profile
- Company to host investor call to discuss interim clinical results and provide a program update on Sunday, June 6 at 6:00 PM ET

Company Conference Call and Webcast Information

Merus will hold a conference call and webcast for investors on Sunday, June 6, 2021 at 6:00 PM ET to discuss the Zeno clinical data. A replay will be available after the completion of the call in the Investors and Media section of our website.

Date: Sunday, June 6 at 6:00 pm ET
Webcast link: available on our website
Dial-in: Toll-Free: 1-877-260-1463 / International: 1-706-643-5907
Conference ID: 9678617

https://finance.yahoo.com/news/merus-presents-clinical-data-zenocutuzumab-160000708.html

#ASCO21: Iovance Clinical Data for Lifileucel Combo in Advanced Melanoma

 86% Overall Response Rate (ORR) and 43% Complete Response Rate in Immune Checkpoint Inhibitor (ICI) Naïve Advanced Melanoma Patients in IOV-COM-202 Clinical Study

Initial 7 Patients Show 3 Complete Responses, 3 Partial Responses and 1 Best Response of Stable Disease

ASCO Update Conference Call and Webcast on Sunday, June 6 at 12 p.m. ET

Webcast and Conference Call
Iovance will host a webcast and conference call on Sunday, June 6, at 12:00 p.m. ET to discuss ASCO clinical data updates for lifileucel alone and in combination with pembrolizumab in patients with advanced melanoma. Iovance senior leadership, together with Dr. Omid Hamid of The Angeles Clinic, will present a summary of the ASCO data from Cohort 1A in the IOV-COM-202 study as well as the upcoming oral presentation of updated Cohort 2 data from the C-144-01 clinical study.

The conference call dial-in numbers are 1-844-646-4465 (domestic) or 1-615-247-0257 (international) and the access code is 4858337. The live webcast can be accessed in the Investors section of the company’s website at http://www.iovance.com. The archived webcast will be available for a year in the Investors section at www.iovance.com.

https://finance.yahoo.com/news/iovance-biotherapeutics-announces-clinical-data-130800517.html

#ASCO21: Puma Bio Presents Data Comparing Findings from Phase II CONTROL Trial with Neratinib Arm of Phase III ExteNET Trial

 Dose escalation of neratinib lowers the frequency of severe diarrhea and improves overall tolerability in patients with HER2-positive early stage breast cancer

 

Puma Biotechnology, Inc. (Nasdaq: PBYI), a biopharmaceutical company, presented results at the virtual 2021 ASCO Annual Meeting comparing the diarrhea mitigation strategies investigated in the Phase II CONTROL trial with the neratinib treatment arm of the ExteNET trial where diarrhea prophylaxis was not required. The presentation, entitled “Dose escalation for mitigating diarrhea: Ranked tolerability assessment of antidiarrheal regimens in patients receiving neratinib for early-stage breast cancer,” is included in the Breast Cancer—Local/Regional/Adjuvant Poster Session (#536).

The CONTROL trial is an international, open-label, Phase II study investigating the use of antidiarrheal prophylaxis or dose escalation to improve the tolerability of neratinib-associated diarrhea. The primary endpoint of the trial is the incidence of grade 3 diarrhea. Patients ≥18 years of age with stage I–IIIc HER2-positive breast cancer received neratinib (240 mg/day orally for 1 year) together with one of the regimens investigated: loperamide alone, in combination with budesonide or colestipol, or neratinib dose escalation (DE): 120 mg/day on days 1–7, 160 mg/day on days 8–14, then 240 mg/day thereafter + loperamide PRN.

In the analysis presented at ASCO 2021, five CONTROL cohorts that had completed follow up were evaluated for 13 endpoints related to tolerability. The DE cohort ranked the best among the CONTROL cohorts and was then compared with the neratinib arm of the ExteNET trial, which included patients ≥18 years of age with stage I–III HER2-positive breast cancer receiving neratinib 240 mg/day or matching placebo for one year, without mandated anti-diarrheal treatment. ExteNET was a multicenter, randomized, double-blind, Phase III trial (NCT00878709) of 2,840 HER2-positive early stage breast cancer patients who received neratinib after neoadjuvant and/or adjuvant therapy with chemotherapy and a trastuzumab-based regimen.

Comparison of the CONTROL DE cohort with the ExteNET neratinib arm demonstrated that grade 3 diarrhea was substantially lower in CONTROL DE compared to ExteNET (13.3% vs. 39.9%). Neratinib DE also resulted in fewer total days of grade 3 diarrhea compared to ExteNET (2.5 days vs 5 days). Dose escalation also led to fewer discontinuations due to diarrhea in the first three months of treatment compared to ExteNET (3.3% vs 14.5%). Additionally, the average duration of treatment with neratinib was much longer in the DE cohort versus ExteNET. Overall, the findings of this analysis suggest that escalating the dose of neratinib in the first 2 weeks of treatment may help patients stay on neratinib longer, allowing them the opportunity to complete the recommended 1-year of treatment.

https://www.biospace.com/article/releases/puma-biotechnology-presents-data-comparing-findings-from-the-phase-ii-control-trial-with-the-neratinib-arm-of-the-phase-iii-extenet-trial-at-the-asco-2021-annual-meeting/