Remove Events Remove Pharmacokinetics Remove Regulations Remove Small Molecule
article thumbnail

Study offers possible treatment for frontotemporal dementia

Drug Discovery World

FTD(GRN), which is estimated to account for a quarter of familial FTD cases, is a form of FTD caused by mutations of the progranulin gene (PGRN), a protein responsible for the regulation of cell growth, survival, and repair.

Treatment 147
article thumbnail

Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

The Pharma Data

LNP023 also demonstrated a favorable safety and tolerability profile with no serious treatment-related infections or thromboembolic events. Following the cut-off date for the presented data, one participant, who had severe lymphopenia at study entry, discontinued treatment due to a serious adverse event (AE) of lymphoproliferative disorder.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Aptorum Group Receives Clearance from Health Canada to Initiate a Phase 1 Clinical Trial for ALS-4, a Small Molecule Drug for Infections Caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)

The Pharma Data

The secondary objective is to assess the pharmacokinetic profile of SAD and MAD of ALS-4 administered orally to healthy subjects. Aptorum Group assumes no obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About ALS-4.

article thumbnail

Aptorum Group Announces Submission of Clinical Trial Application for ALS-4, an Orally Administered Small Molecule Drug for the Treatment of Infections caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)

The Pharma Data

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. ALS-4 is a novel small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.

article thumbnail

Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

LifeSciVC

For KLK5 and KLK7 as well as their endogenous regulator (LEKTI, encoded by SPINK5 ) and one of their substrates (filaggrin, FLG ) there is evidence that KLK5 and 7 up-regulation is pathogenic and down-regulation protective in epidermal barrier dysfunction (especially for Atopic Dermatitis). in liver, in CNS)?

article thumbnail

Analysis Life Sciences Thank You FDA finalizes new guidance on nonclinical assessment of potential immunotoxicity

Agency IQ

However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs. The guidance contains recommendations for small molecule drugs, as well as any biological products that fall within CDER’s purview.

FDA 40
article thumbnail

Aptose Initiates Dosing of CG-806 in Patients with Acute Myeloid LeukemiaPhase 1 a/b Study of CG-806 in AML Initiates with Starting Dose of 450mg

The Pharma Data

Several clinical sites are screening patients for the Phase 1 a/b multicenter, open-label, dose escalation study of safety, pharmacodynamics, and pharmacokinetics of CG-806 in ascending cohorts (3+3 design) to determine the maximum tolerated dose or recommended dose in patients with relapsed or refractory AML. . .