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Milestone Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Regulatory and Corporate Update

Retrieved on:

Thursday, March 21, 2024

MONTREAL and CHARLOTTE, N.C., March 21, 2024 (GLOBE NEWSWIRE) -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST) today reported financial results for the fourth quarter and year ended December 31, 2023 and provided a regulatory and corporate update.

Key Points:

- FDA reiterated prior guidance on regulatory pathway for AFib-RVR, End of Phase 2 Meeting expected mid-2024
MONTREAL and CHARLOTTE, N.C., March 21, 2024 (GLOBE NEWSWIRE) -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST) today reported financial results for the fourth quarter and year ended December 31, 2023 and provided a regulatory and corporate update.
There was no revenue recorded for the fourth quarter of 2023, compared with $3.5 million the fourth quarter of 2022.
Commercial expense for the fourth quarter of 2023 was $5.0 million, compared with $2.6 million for the prior year period.
For the fourth quarter of 2023, net loss was $13.6 million, compared to $13.2 million for the prior year period.

Lisata Therapeutics Announces U.S. FDA Rare Pediatric Disease Designation Granted to LSTA1 for the Treatment of Osteosarcoma

Retrieved on:

Thursday, March 21, 2024

Osteosarcoma, Food and Drug Administration, NDA, Cancer, Therapy, FDA, Food, Pharmaceutical industry

BASKING RIDGE, N.J., March 21, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the “FDA”) has granted Rare Pediatric Disease Designation (“RPDD”) to LSTA1, the Company’s lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults.

Key Points:

BASKING RIDGE, N.J., March 21, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the “FDA”) has granted Rare Pediatric Disease Designation (“RPDD”) to LSTA1, the Company’s lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults.
A substantial benefit of a RPDD is receipt of a priority review voucher, often referred to as a “golden ticket,” once the FDA approves the new drug application (“NDA”) for the product and indication having received the RPDD.
The voucher may be used by the sponsor or sold to another sponsor for their use.
Priority review vouchers have sold for as much as $350 million USD historically and, more recently, have sold for $75 to $100 million USD.

X4 Pharmaceuticals Reports Fourth-Quarter and Full-Year 2023 Financial Results and Provides Corporate Update

Retrieved on:

Thursday, March 21, 2024

BOSTON, March 21, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the fourth quarter and full year ended December 31, 2023, and highlighted key 2023 events and expected upcoming milestones.

Key Points:

“Following an incredibly productive 2023, we are expecting a transformative year in 2024,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights.
The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .
Following the completion of the call, a webcast replay of the conference call will be available on the company website.

Tonix Pharmaceuticals Announces Selection of Two Contract Manufacturing Organizations for the Launch and Commercial Manufacture of Tonmya™ for the Management of Fibromyalgia

Retrieved on:

Wednesday, March 20, 2024

Fibromyalgia, Partnership, Patient, Tablet, Hydrogen chloride, NDA, Tonix Pharmaceuticals, Fatigue, Food, CMOS, Pharmaceutical industry

CHATHAM, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced it has selected two contract manufacturing organizations (CMOs), one of which is Almac Pharma Services, a member of the privately owned Almac Group, as dual supply sources for the potential launch and commercialization of Tonmya™ (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in the U.S.

Key Points:

As previously announced, Tonix’s second positive Phase 3 study, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia.
Statistically significant and clinically meaningful results (p=0.001 or better) were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function.
TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies, and no new safety signals were observed.
Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration in the second half of 2024 for Tonmya for the management of fibromyalgia.

Crinetics’ Once-Daily Oral Paltusotine Achieved the Primary and All Secondary Endpoints in the Phase 3 PATHFNDR-2 Study in Acromegaly Patients

Retrieved on:

Tuesday, March 19, 2024

Arthralgia, Abdominal pain, Acromegaly, Headache, Webcast, Patient, Diarrhea, NDA, FISU World University Games, ID, Endocrinology, Food, Medical school, Pharmaceutical industry

SAN DIEGO, March 19, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors, today announced positive topline results from PATHFNDR-2, the second of two Phase 3 studies evaluating the efficacy and safety of oral, once-daily investigational paltusotine for the treatment of acromegaly.

Key Points:

“This study demonstrates that paltusotine can provide both symptom control as well as biochemical control in patients who are not currently on pharmacologic treatment.
The frequency of participants with at least one treatment emergent adverse event (TEAE) was comparable in the paltusotine treatment arm and placebo arm.
The frequency of adverse events considered related to acromegaly was notably lower in paltusotine treated participants compared to placebo treated participants.
Following the live event, a replay will be available on the Investors section of the Company’s website.

Journey Medical Corporation Announces U.S. FDA Acceptance of New Drug Application for DFD-29 for the Treatment of Rosacea

Retrieved on:

Monday, March 18, 2024

Entrepreneurship, Capsule, Patient, Food and Drug Administration, Rosacea, NDA, Red, Minocycline, Erythema, Food, Marketing, FDA, CEA, Physician, IGA, PDUFA, Pharmaceutical industry

The FDA has set a Prescription Drug User Fee Act (“PDUFA”) goal date of November 4, 2024.

Key Points:

The FDA has set a Prescription Drug User Fee Act (“PDUFA”) goal date of November 4, 2024.
The Phase 3 clinical trials achieved all co-primary and secondary endpoints, and subjects completed the 16-week treatment with no significant safety issues.
DFD-29 demonstrated statistically significant superiority over both the current standard-of-care treatment, Oracea® 40 mg capsules, and placebo for Investigator’s Global Assessment (IGA) treatment success as well as the reduction in the total inflammatory lesion count in both studies.
For the secondary endpoint evaluating erythema (redness) associated with rosacea, DFD-29 showed a statistically significant reduction in Clinician’s Erythema Assessment (CEA) compared to placebo in both clinical trials.”

Eyenovia Reports Fourth Quarter and Full-Year 2023 Financial Results and Provides Business Update

Retrieved on:

Monday, March 18, 2024

Research, Clobetasol, Inflammation, NDA, Co-promotion, Clinical trial, Optometry, FDA, Vision Source, Pharmaceutical industry

NEW YORK, March 18, 2024 (GLOBE NEWSWIRE) -- Eyenovia, Inc. (NASDAQ: EYEN), a commercial-stage ophthalmic company, today announced its financial and operating results for the fourth quarter and full-year ended December 31, 2023.

Key Points:

For the fourth quarter of 2023, net loss was approximately $8.0 million, or $0.18 per share.
Research and development expenses totaled approximately $4.1 million for the fourth quarter of 2023, compared to $2.2 million for the fourth quarter of 2022, an increase of approximately 84.6%.
For the fourth quarter of 2023, general and administrative expenses were approximately $3.4 million, compared to $3.2 million for the fourth quarter of 2022, an increase of approximately 7.3%.
Total operating expenses for the fourth quarter of 2023 were approximately $7.5 million, compared to approximately $5.4 million for the fourth quarter of 2022.

Oculis Reports Q4 and Full Year 2023 Financial Results and Update on Company Progress

Retrieved on:

Monday, March 18, 2024

Riad Sherif M.D., Chief Executive Officer of Oculis: “2023 was a remarkable milestone-rich year for Oculis.

Key Points:

Riad Sherif M.D., Chief Executive Officer of Oculis: “2023 was a remarkable milestone-rich year for Oculis.
These non-IFRS financial measures exclude the impact of items that the Company’s management believes affect comparability or underlying business trends.
The Company’s management uses these measures to better analyze its financial results and better estimate its financial outlook.
The resulting non-cash foreign exchange impact of such reclassification amounted to CHF 5.0 million or $5.7 million for the year ended December 31, 2023.

Aquestive Therapeutics Announces Pivotal Study for Anaphylm™ (epinephrine) Sublingual Film Successfully Meets Primary and Secondary Endpoints and Provides Clinical Development Update Following FDA Meeting

Retrieved on:

Thursday, March 14, 2024

“We are extremely pleased with the pivotal study results as well as our recent FDA interaction,” said Daniel Barber, President and Chief Executive Officer of Aquestive.

Key Points:

“We are extremely pleased with the pivotal study results as well as our recent FDA interaction,” said Daniel Barber, President and Chief Executive Officer of Aquestive.
“We believe we have a clear understanding of the remaining clinical development steps necessary for a pre-NDA meeting with the FDA in the second half of the year.
These latest study results show that the sublingual administration of epinephrine provides rapid and sustained levels of epinephrine similar to approved treatments.
The FDA noted that substantial progress had been made in the Anaphylm clinical development program and did not outline any new clinical development requirements.

IgA Nephropathy Pipeline Dynamics Signal Further Treatment Evolution on the Horizon

Retrieved on:

Thursday, March 14, 2024

For several years, Spherix Global Insights has been closely tracking the IgAN market and recently released its fourth annual US patient chart audit study.

Key Points:

For several years, Spherix Global Insights has been closely tracking the IgAN market and recently released its fourth annual US patient chart audit study.
Spherix is the leading authority on IgAN market dynamics and will continue to provide insight into how nephrologists are treating and approaching care for their IgAN patients.
Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits.
Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns.