Karyopharm, Menarini Group receive positive CHMP opinion for NEXPOVIO » 08:4705/2005/20/22
Karyopharm Therapeutics and the Menarini Group, a privately-held, international pharmaceutical company, announced that the European Medicines Agency's Committee for Medicinal Products for Human Use has adopted a positive opinion recommending approval of NEXPOVIO, a first-in-class, oral exportin 1 inhibitor, in combination with once weekly bortezomib and low-dose dexamethasone for the treatment of adults with multiple myeloma who have received one to three prior lines of therapy. The positive CHMP opinion is a scientific recommendation for marketing authorization and one of the final steps before the European Commission makes a decision on Karyopharm's NEXPOVIO application. The EC's decision is expected within approximately 60 days following the CHMP recommendation. In December 2021, Karyopharm and Menarini Group entered into an exclusive license agreement to commercialize NEXPOVIO in Europe.
Benchmark says Emergent, Siga, Chimerix strength attributable to monkeypox scare » 08:3805/2005/20/22
EBS, SIGA, CMRX
Benchmark analyst Robert…
Kymera presents preclinical data on IRAK4 degrader KT-474 at SID meeting » 07:3505/2005/20/22
BofA starts TG Therapeutics at Underperform, sees difficult commercial setup » 07:0605/2005/20/22
BofA analyst Alec…
BofA analyst Alec Stranahan initiated coverage of TG Therapeutics with an Underperform rating and $5.00 price target. The stock is down 69% year-to-date since the announcement of voluntary withdrawals of U2 BLA and the terminated commercialization of Ukoniq, but ublituximab still faces 'difficult' commercial setup, the analyst tells investors in a research note. Stranahan adds that discounted pricing for the drug would be difficult as TG Therapeutics "struggles with cash", and the drug's one-hour infusion time is not a strong case for convenience.
TG Therapeutics initiated with an Underperform at BofA » 06:1305/2005/20/22
BofA analyst Alec…
BofA analyst Alec Stranahan initiated coverage of TG Therapeutics with an Underperform rating and $5 price target.
Mustang Bio announces updated interim data on XSCID treatment » 08:5705/1905/19/22
Mustang Bio announced…
Mustang Bio announced that interim Phase 1/2 data on treatment with the same lentiviral vector used in MB-107, Mustang's lentiviral gene therapy for X-linked severe combined immunodeficiency, also known as bubble boy disease, in newly diagnosed infants under the age of two, support plans to initiate a multicenter pivotal Phase 2 trial for MB-107 under the Company's Investigational New Drug application in the second half of this year. Representing the largest cohort of infants with XSCID treated with gene therapy, the data include 23 infants with XSCID treated with the lentiviral vector at a median age of 3 months with a median follow-up of 2.6 years. Transduced autologous bone marrow CD34+ cells were generated for all patients with a median vector copy number of 0.81/cell and a median CD34+ cell dose of 9.61x106/kg. Prior to the infusion of cells, patients received busulfan targeted to a cumulative area-under-the-curve of 22 mg*hr/L. The treatment was well tolerated, and all patients experienced complete hematopoietic recovery. Severe adverse events occurred in three patients and all resolved. Seventeen patients had active infections prior to therapy, and in all cases these infections cleared. In addition, 15 patients have so far been able to discontinue intravenous immunoglobulin, and to date 14 patients have been successfully immunized. All patients are currently alive with stable vector marking in all cell lineages and without evidence of malignant transformation. The data, presented orally during the Clinical Trials Spotlight Symposium at the 25th Annual Meeting of the American Society of Gene & Cell Therapy, are from a multicenter Phase 1/2 clinical trial underway at St. Jude, UCSF Benioff Children's Hospital in San Francisco and Seattle Children's Hospital. The lentiviral gene therapy is also being assessed in a Phase 1/2 clinical trial at the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, for XSCID patients who have been previously treated with hematopoietic stem cell transplantation and for whom re-treatment is indicated.
Allogene Therapeutics presents preclinical data on allogeneic CAR T cells » 08:4805/1905/19/22
Allogene Therapeutics will present preclinical findings evaluating the characteristics and function of donor-derived allogeneic CAR T cells. Data showed that cells from a diverse set of younger donors had improved characteristics and better in vitro anti-tumor activity compared to cells from older donors. The study also showed that cells from patients with certain cancers generally performed suboptimally based on functional assays and often could not be used to generate viable CAR T therapies. The findings will be presented during an oral session at the 2022 American Society of Gene and Cell Therapy Annual Meeting at 10:45am ET. The study evaluated the characteristics and performance of CAR T cells derived from healthy donors aged 19 to 62, comparing the healthy donor cells to those derived from patients with cancer. Based on the analysis, CAR T cells produced from younger donors had stronger T cell phenotypes and better in vitro anti-tumor activity cytotoxicity compared to older donors. The expression of specific exhaustion and activation markers was also correlated with increased donor age and in vitro anti-tumor activity decreased with donor age. Regardless of age, the CAR T cells derived from healthy donors performed better and had a lower manufacturing failure rate compared to those derived from patients with cancer. Creating allogeneic CAR T cells from healthy donors reduces product variability; reduces the risk of manufacturing failures; and enables treatment within days, eliminating the need for bridging chemotherapy. This study provides additional evidence that younger, healthy donors may improve product characteristics and potency compared to older donors. Allogene currently has four clinical programs underway investigating the potential of AlloCAR T product candidates for the treatment of relapsed/refractory large B cell lymphoma, RR multiple myeloma and advanced renal cell carcinoma.
X4 Pharmaceuticals price target lowered to $7 from $10 at B. Riley » 08:3805/1905/19/22
B. Riley analyst Mayank…
B. Riley analyst Mayank Mamtani lowered the firm's price target on X4 Pharmaceuticals to $7 from $10 and keeps a Buy rating on the shares. While management's ability to address the longstanding balance sheet overhang remains a challenge, partly contributing to equity underperformance year-to-date, equity trades way below cash and heavily discounts the likelihood of success for three data catalysts and subsequent capital-efficient drug launch beginning in 2023, Mamtani tells investors in a research note.
Maxim biotechnology analysts to hold an analyst/industry conference call » 04:5505/1905/19/22
Biotechnology Analysts, along with Dr. Sabine Mueller a pediatric neuro-oncologist at the UCSF Brain Tumor Center, holds a panel discussion on new targets for pediatric brain cancer on an Analyst/Industry conference call on May 19.
Scilex initiates Phase 2 study to evaluate safety, efficacy of SP-103 » 09:3305/1805/18/22
Scilex Holding Company…
Scilex Holding Company announced dosing of the first subject in a Phase 2, randomized, double-blind, placebo-controlled, parallel group, multicenter study to evaluate the safety and efficacy of SP-103 in subjects with acute LBP. Scilex's current marketed product, ZTlido, has label claims regarding superior adhesion qualities as compared to other products and SP-103 has the same adhesion characteristics. Scilex is developing SP-103 to be a triple-strength, non-aqueous lidocaine topical system for the treatment of acute LBP. Acute LBP can range in intensity from a dull, constant ache to a sudden, sharp sensation that leaves the person incapacitated. It is estimated that approximately 65 million adults in the U.S., or 25% of U.S. adults, suffer from acute back pain with a total potential global market opportunity of approximately $10.0 billion by 2026.