New Drug Application

Crossject announces highly successful closing of its €8 million rights offering

Retrieved on: 
Tuesday, June 4, 2024

Dijon, France 4 April, 2024 -- Crossject (ISIN: FR0011716265; Euronext: ALCJ), a specialty pharma company developing needle-free auto-injectors for emergency situations, announces the successful closing of its rights offering with maintenance of preferential subscription rights for a gross amount of approximately €8 million as announced on 30 April 2024. The net proceeds from this round are approximately €7.6 million. This round of financing is an important step to continue to support Crossject’s activities in bringing its ZEPIZURE® emergency epilepsy treatment to market and in building its U.S. operations in anticipation of its direct commercialization. The capital increase was at a subscription price of € 1.848, representing a discount of 10% to the closing price on 30 April 2024.

Key Points: 
  • The capital increase was at a subscription price of € 1.848, representing a discount of 10% to the closing price on 30 April 2024.
  • All the demand fulfilled on an irreducible or reducible basis was enough to meet our objectives.
  • With its ambition to directly commercialize ZEPIZURE® in the U.S., Crossject has entered into a new phase in its development as a specialty pharmaceuticals company.
  • As of the date of this press release and after the offering, Gemmes Venture holds an unchanged 24.48% of the share capital.

UroGen Announces Unprecedented 82.3% Duration of Response at 12 Months in the ENVISION Trial Investigating UGN-102 as Potentially the First FDA-Approved Non-Surgical Treatment for LG-IR-NMIBC

Retrieved on: 
Thursday, June 13, 2024

The ENVISION trial previously met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months following the first instillation of UGN-102.

Key Points: 
  • The ENVISION trial previously met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months following the first instillation of UGN-102.
  • The ENVISION Phase 3 study is investigating UGN-102 in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
  • In addition to the 12-month data, the DOR Kaplan Meier estimates at 15 (n=43) and 18 (n=9) months were both 80.9% (95%CI, 73.9%, 86.2%).
  • The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.

TAGRISSO® (osimertinib) granted Priority Review in the US for patients with unresectable, Stage III EGFR-mutated lung cancer

Retrieved on: 
Monday, June 10, 2024

If approved, TAGRISSO will be indicated for EGFRm patients whose tumors have exon 19 deletions or exon 21 (L858R) mutations.

Key Points: 
  • If approved, TAGRISSO will be indicated for EGFRm patients whose tumors have exon 19 deletions or exon 21 (L858R) mutations.
  • Median progression-free survival (PFS) was 39.1 months in patients treated with TAGRISSO versus 5.6 months for placebo.
  • Interstitial lung disease (ILD)/pneumonitis occurred in 4% of the 1813 TAGRISSO-treated patients; 0.4% of cases were fatal.
  • AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

FDA Approves Almirall’s Klisyri® (tirbanibulin) for the Treatment of Actinic Keratosis on Expanded Area of Face or Scalp up to 100 cm2

Retrieved on: 
Monday, June 10, 2024

Klisyri, a microtubule inhibitor ointment, is now approved in a 350 mg package size and is a 5-day topical field treatment for actinic keratosis (AK) of the face or scalp.

Key Points: 
  • Klisyri, a microtubule inhibitor ointment, is now approved in a 350 mg package size and is a 5-day topical field treatment for actinic keratosis (AK) of the face or scalp.
  • This new approval will change the previous Klisyri (tirbanibulin) dosing for surface area treatment from up to 25 cm2 to up to 100 cm2, allowing clinicians to treat a larger area of the face or balding scalp.
  • The sNDA was supported by an additional Phase 3, multicenter, open-label, clinical safety study with more than 100 patients in the US.
  • The most common adverse reactions (incidence ≥2%) were local skin reactions, application site pruritus, and application site pain.

Dr. Giovanni Selvaggi, Who Has Brought Several Oncology Drugs to Market, Joins CEL-SCI as Clinical Advisor

Retrieved on: 
Thursday, June 6, 2024

CEL-SCI Corporation (NYSE American: CVM) today announced Dr. Giovanni Selvaggi, an oncology key opinion leader instrumental in successfully bringing several drugs to market has joined CEL-SCI as a Clinical Advisor.

Key Points: 
  • CEL-SCI Corporation (NYSE American: CVM) today announced Dr. Giovanni Selvaggi, an oncology key opinion leader instrumental in successfully bringing several drugs to market has joined CEL-SCI as a Clinical Advisor.
  • View the full release here: https://www.businesswire.com/news/home/20240606671425/en/
    Multikine is the first investigational pre-surgical cancer drug in newly diagnosed head and neck cancer.
  • He is also a Clinical Consultant to Tubulis GmBH for a first-in-class ADC program in solid tumors, and Clinical Strategy Senior Advisor to Alira Health.
  • Selvaggi has a passion for bringing cancer drugs to market to save lives and lead clinical research toward a cure for cancer.

Precision BioSciences Presents New Preclinical Safety Data for PBGENE-HBV Clinical Candidate at the European Association for the Study of the Liver Congress

Retrieved on: 
Wednesday, June 5, 2024

The poster will also showcase non-human primate data demonstrating good tolerability of a multi-dosing approach for the treatment of chronic hepatitis B.

Key Points: 
  • The poster will also showcase non-human primate data demonstrating good tolerability of a multi-dosing approach for the treatment of chronic hepatitis B.
  • “We are pleased to present new safety data at EASL for our lead PBGENE-HBV program.
  • Preclinical safety data supports the advancement of PBGENE-HBV to clinical trials as a potentially curative treatment for chronic hepatitis B.
  • Full poster details are included below:
    Presenter: Emily Harrison, PhD, Senior Scientist - Hepatitis Research Leader, Precision BioSciences

District Court Issues Favorable Ruling and Denies United Therapeutics’ Request to Block YUTREPIA™ Launch

Retrieved on: 
Monday, June 3, 2024

The ruling reinforces the clear path for the U.S. Food and Drug Administration (FDA) to issue a final decision on the amended New Drug Application (NDA) for YUTREPIA.

Key Points: 
  • The ruling reinforces the clear path for the U.S. Food and Drug Administration (FDA) to issue a final decision on the amended New Drug Application (NDA) for YUTREPIA.
  • 23-975) filed by UTHR in September 2023 in which it has alleged YUTREPIA would infringe U.S. Patent No.
  • UTHR has stated that it plans to appeal the Federal Circuit’s affirmation of the invalidity of the ‘793 Patent to the United States Supreme Court.
  • Separately, UTHR has also appealed Judge Andrews’ ruling to set aside an injunction that he had previously issued blocking the launch of YUTREPIA based solely on the ‘793 Patent.

Iterum Therapeutics Receives FDA Acceptance of Resubmission of NDA for Oral Sulopenem for the treatment of Uncomplicated Urinary Tract Infections

Retrieved on: 
Friday, May 31, 2024

Under the Prescription Drug User Fee Act (“PDUFA”), the FDA has deemed the Company’s NDA resubmission to be a Class II complete response which has a six-month review period from the date of resubmission.

Key Points: 
  • Under the Prescription Drug User Fee Act (“PDUFA”), the FDA has deemed the Company’s NDA resubmission to be a Class II complete response which has a six-month review period from the date of resubmission.
  • As a result, the FDA has assigned a PDUFA action date of October 25, 2024.
  • “We are very pleased that the FDA has accepted the resubmission of the NDA for oral sulopenem,” said Corey Fishman, Chief Executive Officer.
  • “This significant milestone brings us one step closer to the potential approval of oral sulopenem for uUTIs providing patients and physicians with a much-needed treatment option for this underserved market.

Larimar Therapeutics Selected by FDA to Participate in START Pilot Program for Nomlabofusp in Friedreich’s Ataxia

Retrieved on: 
Thursday, May 30, 2024

BALA CYNWYD, Pa., May 30, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that the United States Food and Drug Administration (FDA) has selected nomlabofusp to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.

Key Points: 
  • BALA CYNWYD, Pa., May 30, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that the United States Food and Drug Administration (FDA) has selected nomlabofusp to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.
  • Nomlabofusp is a novel protein replacement therapy designed to address the root cause of Friedreich’s ataxia (FA) by delivering frataxin to mitochondria.
  • “We are thrilled nomlabofusp, the first potential therapy to increase frataxin levels in patients with FA, has been selected by the FDA to participate in the START pilot program.
  • “We look forward to participating in this pilot program and working with the FDA to further the development of nomlabofusp which has the potential to address the root cause of Friedreich’s Ataxia by increasing frataxin levels.”
    Dr. Russell Clayton, Chief Medical Officer of Larimar added, “Participating in the START program enables increased communication with the FDA to help expedite the progression of our nomlabofusp development program to the pre-BLA meeting stage.

Catalyst Pharmaceuticals Receives U.S. FDA Approval For Increased Maximum Daily Dose For FIRDAPSE®

Retrieved on: 
Thursday, May 30, 2024

The increased maximum daily dose offers healthcare providers and patients greater flexibility in treatment regimens for the management of LEMS.

Key Points: 
  • The increased maximum daily dose offers healthcare providers and patients greater flexibility in treatment regimens for the management of LEMS.
  • FIRDAPSE is currently the only U.S. approved treatment for LEMS and this approval broadens the approved dosing options for prescribers treating LEMS.
  • “We are pleased to receive the approval for the increased maximum daily dose of FIRDAPSE,” said Richard J. Daly, President and CEO of Catalyst.
  • “This pivotal achievement further underscores our dedication to meeting the evolving needs of LEMS patients and their healthcare providers.