logo
  

BLCM Hit With Clinical Hold, LANTERN Fails To Light Up LYRA, TNXP Gains, FDA Says No To SCPH

biptech dec08 lt

Today's Daily Dose brings you news about the clinical hold imposed on Bellicum Pharma's BPX-601 trial, PDUFA date of Chimerix' New Drug Application for Brincidofovir as a medical countermeasure for smallpox, Gamida's phase I clinical study of GDA-201 in patients with non-Hodgkin lymphoma and multiple myeloma, Oncternal's promising data related to Cirmtuzumab in relapsed/refractory mantle cell lymphoma and chronic lymphocytic leukemia, Lyra's LANTERN study data, and FDA refusing to approve scPharma's drug-device for edema.

Read on…

1. FDA Slaps Clinical Hold on Bellicum's BPX-601 Study

The FDA has placed a clinical hold on Bellicum Pharmaceuticals Inc.'s (BLCM) ongoing phase I/II dose-escalation trial of BPX-601 in patients with previously treated metastatic pancreatic or prostate cancer, following the death of a pancreatic cancer patient in the study.

The patient death has been classified as unrelated to BPX-601 by the company.

The clinical hold does not affect the company's plans to initiate enrollment in the phase I/II clinical trial of BPX-603, a dual switch GoCAR-T, in patients with HER2+ solid tumors by the end of the year.

BLCM closed Monday's trading at $2.79, down 9.71%.

2. Chimerix to face FDA in April

Shares of Chimerix Inc. (CMRX) touched a new 52-week high of $4.58 on Monday, thanks to the update on the company's New Drug Application for Brincidofovir as a medical countermeasure for smallpox.

Brincidofovir has been accepted for priority review by the FDA, with a decision expected on April 7, 2021. The FDA is not planning to hold an advisory committee meeting to discuss the Brincidofovir application.

Chimerix developed Brincidofovir under an ongoing collaboration and funding provided by the Biomedical Advanced Research and Development Authority (BARDA).

The company ended September 30, 2020 with $87.8 million of capital available to fund operations, no debt, and approximately 62.6 million outstanding shares of common stock.

CMRX closed Monday's trading at $4.54, up 25.41%.

3. Gamida Catches Investors' Eyes

Shares of Gamida Cell Ltd. (GMDA) jumped more than 50% on Monday, following presentation of the updated and expanded results from a phase I clinical study of GDA-201 in patients with non-Hodgkin lymphoma (NHL) and multiple myeloma at the American Society of Hematology (ASH) Annual Meeting & Exposition.

According to the trial results, GDA-201 was well-tolerated and no dose-limiting toxicities in 35 patients (19 with NHL and 16 with MM).

GDA-201 resulted in significant clinical activity in heavily pretreated patients with advanced non-Hodgkin lymphoma. Of the 19 patients with NHL, 13 complete responses and one partial response were observed, with an overall response rate of 74 percent and a complete response rate of 68 percent.

The maximum tolerated dose was not achieved, as no dose limiting toxicities were observed in patients who received the maximum target dose.

GMDA closed Monday's trading at $10.89, up 56.24%.

4. Oncternal Marches Ahead with Cirmtuzumab Data

Shares of Oncternal Therapeutics Inc. (ONCT) surged nearly 50% on Monday, following an update on interim clinical data from the company's ongoing phase I/II trial of Cirmtuzumab in combination with Ibrutinib in patients with relapsed/refractory mantle cell lymphoma and chronic lymphocytic leukemia.

According to the trial results, the best objective response rate of 87% was observed for 15 patients with relapsed/refractory mantle cell lymphoma (MCL), with a median follow-up of 12.1 months. Median progression-free survival (PFS) was not reached.

The best objective response rate was 83% at the time when the company presented the data at the American Society of Clinical Oncology (ASCO) 2020 Virtual Annual Meeting in May.

As for the 49 chronic lymphocytic leukemia (CLL) patients in the trial, the overall best objective response rate was 92%. The median PFS was not reached for patients with treatment-naïve CLL after a median follow-up of 16.6 months, and median PFS was 29.5 months for patients with r/r CLL after a median follow-up of 17.1 months.

The overall best objective response rate was 88% for the CLL patients in May.

Commenting on the results, James Breitmeyer, Oncternal's President and CEO, said, "We are pleased that median PFS has not yet been reached after a median follow-up of over 12 months in the MCL patients, and are encouraged that both PFS and ORR have improved with longer follow-up. We are in active dialogue with FDA on pivotal study design in order to define the path to approval in MCL."

ONCT closed Monday's trading at $3.98, up 49.62%.

5. LANTERN Fails To Light Up Lyra Therapeutics

Shares of Lyra Therapeutics Inc. (LYRA) plunged more than 25% on Monday, hurt by the failure of the company's LANTERN study to meet the primary endpoint.

LANTERN is a phase II study of LYR-210 in adult patients with chronic rhinosinusitis with and without nasal polyps. The change from baseline (CFBL) in average daily nasal blockage/obstruction/congestion (CS7DA4S) score at Week 4 was set as the primary endpoint of the trial.

The multi-center study enrolled 67 patients at sites in Europe, New Zealand and Australia who were randomized into three groups - 7500 mcg (21 patients) or 2500 mcg (23 patients) of LYR-210 , or control (23 patients).

According to the trial results, 7500 mcg dose of LYR-210 did not achieve the primary endpoint at week 4, but achieved statistically significant improvement in a composite score of the 4 cardinal symptoms (4CS) of chronic rhinosinusitis (CRS) in favor of the treatment arm at weeks 16. The 4CS of CRS are nasal blockage, post-nasal discharge, facial pain/pressure, and decreased sense of smell.

Furthermore, the 7500 mcg dose of LYR-210 achieved statistically significant improvement in SNOT-22 score in favor of the treatment arm at weeks 8, 16, 20 and 24. Sinonasal outcome (SNOT-22) score measures the overall impact of chronic rhinosinusitis on patients' quality of life.

LYRA closed Monday's trading at $9.32, down 25.26%.

. FDA Says "NO" To scPharma's Drug-device for Edema Again

scPharmaceuticals Inc.'s (SCPH) FUROSCIX has been refused FDA approval yet again. The company has been seeking approval of Furoscix for the treatment of edema, or fluid overload, in patients with heart failure.

Furoscix is a drug-device combination product consisting of a proprietary Furosemide solution formulated to a neutral pH to allow for subcutaneous infusion via a patented Infusor, a wearable, pre-programmed drug delivery system that is applied to the abdomen for subcutaneous drug administration.

In its Complete Response Letter, the FDA cited their need to conduct pre-approval inspections at two of the company's third-party manufacturing facilities that could not be conducted due to COVID-19 travel restrictions.

The regulatory agency has also raised questions related to testing, labeling, and features of the combination product unrelated to the drug constituent. In addition, the FDA indicated that there were deficiencies at the third-party facility where the company's off-the-shelf alcohol swabs are manufactured.

In June 2018, when the FDA refused approval for Furoscix, it had asked the company to conduct additional human factors studies, device modifications, and potentially a clinical validation study, before the product could be approved.

SCPH closed Monday's trading at $5.62, down 35.64%.

7. Great RELIEF for Tonix Pharma

Tonix Pharmaceuticals Holding Corp.'s (TNXP) phase III trial of TNX-102 SL has met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo in participants with fibromyalgia.

In the study, dubbed RELIEF, the new 5.6 mg dose of TNX-102 SL achieved statistically significant pain reduction over placebo at Week 14, which was the primary endpoint. TNX-102 SL also showed activity in key secondary endpoints of improving sleep and reducing fatigue.

Another potential pivotal phase III study of TNX-102 SL for fibromyalgia, dubbed RALLY, is ongoing, with topline data expected in the second half of 2021.

TNXP closed Monday's trading at $0.70, down 11.11%.

8. Stocks That Hit New Highs/lows

Fate Therapeutics Inc. (FATE) closed Monday's trading at an all-time high of $83.77, up 37.80%.

Codiak BioSciences Inc. (CDAK) closed at an all-time high of $19.98, up 14.50%.

BioCryst Pharmaceuticals Inc. (BCRX) closed at a 52-week high of $7.22, up 18.36%.

Maravai LifeSciences Holdings Inc. (MRVI) closed at an all-time low of $26.82, down 2.90%.

Lyra Therapeutics, Inc. (LYRA) closed at an all-time low of $9.32, down 25.26%.

For comments and feedback contact: editorial@rttnews.com

Business News

Follow RTT