ProQR Therapeutics N.V. announced the first patient dosed in the Phase 2/3 ILLUMINATE clinical trial for sepofarsen in patients with Leber’s Congenital Amaurosis 10. Data from the study are expected at year-end 2020. “The start of the ILLUMINATE trial marks an important milestone towards our goal of bringing this novel and most advanced therapy for LCA10 to patients,” commented Dr. Aniz Girach, Ophthalmologist and Chief Medical Officer of ProQR. “With the ongoing trials in LCA10 and Ushers syndrome type 2, and the start of our clinical program for adRP later this year, we are breaking new ground and paving the way for new treatments in multiple severe inherited retinal diseases.” ILLUMINATE, or PQ-110-003, is a randomized, prospective, double-masked, sham-controlled 24-month trial of sepofarsen that will initially enroll 30 adults and children who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene and a baseline best corrected visual acuity of 3.0 LogMAR or better. The trial is designed with the potential to serve as the sole registration trial for the program. Participants will be assigned equally to three parallel study arms; and a sham control arm with ten participants in each arm. Participants will receive a dose of sepofarsen or a sham-procedure at the start of the trial, at three months and then every six months afterwards. The primary endpoint will be mean change in BCVA from baseline in the active treated arms compared to the control arm.
Monday, April 15, 2019
Intra-Cellular presents additional results from lumateperone study at SIRS
Intra-Cellular announced additional results from its open-label safety switching study assessing the effects of long-term administration of lumateperone, an investigational drug, in patients with stable symptoms of schizophrenia. Results were also presented on the improvements in symptoms of depression in patients with moderate to severe co-morbid depression. The data were presented at the Schizophrenia International Research Society, or SIRS. Study 303 was conducted to assess the long-term effects of treatment with lumateperone on weight and other safety parameters and to observe the impact of switching from standard-of-care, or SOC, antipsychotic medications. The poster presented at SIRS provided additional detail on the initial results presented last year showing that lumateperone, administered for up to one year, was generally well tolerated and exhibited statistically significant improvements from baseline on key safety measures of body weight, cardiometabolic and endocrine parameters, without motor side effects often associated with other antipsychotic medications. In addition, patients treated with lumateperone remained stable with respect to their symptoms of schizophrenia upon switching from SOC. Based on its pharmacology and prior clinical results we believe lumateperone may provide advantages in the treatment of the broad symptoms associated with schizophrenia including depressive symptoms. Thus, additional objectives of the study included determining the effectiveness of lumateperone at treating depressive symptoms in patients with co-morbid depression. The antidepressant effects of lumateperone were assessed using the Calgary Depression Scale for Schizophrenia,or CDSS. In patients with moderate-to-severe depression symptoms at baseline, lumateperone treatment was associated with marked improvement in CDSS scores. Specifically, mean CDSS scores decreased by approximately 60% from 7.4 to 3.1. In addition, 60% of patients met CDSS response criteria by Day 75 and this response rate was maintained through Day 300. Importantly, a similar magnitude of CDSS improvement was seen regardless of concurrent antidepressant therapy.
Genfit data show CymaBay may be differentiated, says Cantor Fitzgerald
Further competitor primary biliary cholangitis data from Genfit’s (GNFTF) elafibranor suggest CymaBay Therapeutics’ (CBAY) seladelpar may have a differentiated profile, Cantor Fitzgerald analyst Eliana Merle tells investors in a research note. The analyst keeps an Overweight rating on CymaBay with a $20 price target.
Biohaven Pharmaceutical price target raised to $89 from $46 at Canaccord
Canaccord analyst Sumant Kulkarni raised his price target on Biohaven Pharmaceutical to $89 from $46 after Bloomberg said the company is exploring a sale. In a research note to investors, Kulkarni, who maintains a Buy rating, says he believes there is significant scarcity value associated with central nervous system-focused companies that have “ripe” pipeline assets, and continues to believe rimegepant, Biohaven’s migraine-focused lead product, could realize its potential more fully in the hands of a larger organization that is able to more effectively target primary care. He continues to like the Biohaven stock on a fundamental basis.
https://thefly.com/landingPageNews.php?id=2892385
https://thefly.com/landingPageNews.php?id=2892385
ADMA Biologics price target raised to $13 from $10 at H.C. Wainwright
H.C. Wainwright analyst Raghuram raised his price target for ADMA Biologics to $13 from $10 following FDA approval of RI-002. Further, the analyst continues to expect near-term feedback from the FDA on ADMA’s response to the Complete Response Letter for Bivigam that the FDA issued in late December 2018. The approval of RI-002 should have favorable implications for the approval of the Bivigam, says the analyst. He reiterates a Buy rating on ADMA Biologics.
https://thefly.com/landingPageNews.php?id=2892395
https://thefly.com/landingPageNews.php?id=2892395
TG Therapeutics granted orphan drug designation for umbralisib by FDA
TG Therapeutics announced that the FDA granted orphan drug designation to its-3-kinase, or PI3K, delta inhibitor, umbralisib, for the treatment of patients with all three types of marginal zone lymphoma, or MZL Umbralisib monotherapy is being evaluated in the UNITY-NHL Phase 2b registration directed clinical trial. The MZL cohort of the UNITY-NHL trial is currently evaluating the safety and efficacy of single agent umbralisib in patients with MZL who have received at least one prior anti-CD20 regimen, the same indication for which the FDA recently granted breakthrough therapy designation for umbralisib.
https://thefly.com/landingPageNews.php?id=2892411
https://thefly.com/landingPageNews.php?id=2892411
Assembly Biosciences to hold a conference call
Conference call to discuss Interim Phase 2a results of ABI-H0731, as presented at the Annual Meeting, will be held on April 15 at 8 am.
Subscribe to:
Posts (Atom)