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Tuesday, March 21, 2023

Gilead CAR T-cell Therapy Improved Overall Survival In Pretreated Lymphoma

 

  • Kite, a Gilead Science Inc  company, announced the primary overall survival (OS) analysis results of the Phase 3 ZUMA-7 study. 
  • The results showed a statistically significant improvement for Yescarta in OS versus historical treatment, which was the standard of care (SOC) in a curative setting for nearly 30 years, for initial treatment of adult patients with relapsed/refractory large B-cell lymphoma (R/R LBCL) within 12 months of completion of first-line therapy. 
  • Historical SOC is a multi-step process involving a platinum-based salvage combination chemoimmunotherapy regimen followed by high-dose therapy (HDT) and a stem cell transplant (ASCT) in those who respond to salvage chemotherapy. 
  • ZUMA-7 was conducted under a Special Protocol Assessment (SPA) with the FDA, whereby the trial design, clinical endpoints, and statistical analysis were agreed upon in advance with the Agency. Other health authorities also agreed with this pre-specified analysis.
  • ZUMA-7 is considered a landmark trial as the first and largest Phase 3 study of any CAR T-cell therapy, with the longest follow-up, which has demonstrated event-free survival (EFS), the primary endpoint, that is superior to historical SOC treatment. 
  • Data from the ZUMA-7 pivotal trial led to the U.S. approval for the initial treatment of R/R LBCL in April 2022 and European Union approval in October 2022.

Coya Has Proof-of-Concept Data From Small Study With ALS Patients

 

  • Coya Therapeutics Inc  reported 48-week clinical data for its proof-of-concept open-label study in 4 Amyotrophic Lateral Sclerosis (ALS) patients indicating that treatment with COYA 302 appeared to ameliorate disease progression.
  • During the 48-week treatment period, COYA 302 appeared to be well tolerated. 
  • Preliminary efficacy of COYA 302 was measured by the ALSFRS-R scale, a validated rating tool for monitoring the progression of disability in ALS patients.
  • The mean (±SD) ALSFRS-R scores at week 24 (33.75 ±3.3) and week 48 (32 ±7.8) after initiation of COYA 302 treatment were not statistically different compared to the ALSFRS-R score at baseline (33.5 ±5.9), indicating significant amelioration in the progression of the disease over the 48-week treatment period.
  • Treg suppressive function, expressed as a percentage of inhibition of proinflammatory T cell proliferation, showed a statistically significant increase over the treatment period and was significantly reduced at the end of the 8-week washout post-treatment period. 
  • The study also evaluated serum biomarkers of inflammation, oxidative stress, and lipid peroxides. 
  • The available data up to 16 weeks suggest a decrease of these biomarker levels, which is consistent with the observed enhancement of Treg function. 
  • The evaluation of the complete biomarker data is ongoing.
  • The company said it plans to file an IND with the FDA in the second half of 2023 and initiate a clinical study soon after.

Yellen Tries To Reassure World That "The US Banking System Remains Sound"

 Watch Treasury Secretary Janet Yellen will address bank leaders from across the country at the American Bankers Association's annual Washington Summit (due to start at 1000ET): 

Read Yellen's prepared remarks below: (emphasis ours)

Altimmune: Interim Phase 2 Obesity Trial and 12-Week Phase 1b Type 2 Diabetes Safety Trial

At Week 24, subjects receiving pemvidutide achieved mean weight losses of 7.3%, 9.4% and 10.7% at the 1.2 mg, 1.8 mg, and 2.4 mg doses, respectively, with the placebo group experiencing a mean weight loss of 1.0% (p < 0.001 at all three doses vs placebo, efficacy estimand using a mixed model of repeated measures [MMRM] analysis). An impact of baseline body weight was observed, where subjects with baseline body weight less than or equal to 115 kg (75% of the study population) achieved mean weight losses of 8.2%, 10.6%, 11.9% and 0.8% at the 1.2 mg, 1.8 mg, 2.4 mg and placebo groups, respectively (p < 0.001 at all three doses vs placebo). Approximately 50% of subjects achieved 10% or more weight loss and approximately 20% of subjects achieved 15% or more weight loss at Week 24 at the 1.8 mg and 2.4 mg doses. Robust reductions in waist circumference (a measure of visceral fat) and serum lipids were also observed, and clinically meaningful reductions in blood pressure were achieved without meaningful increases in heart rate. Glucose homeostasis was also maintained, with no significant changes in fasting glucose or HbA1c.

Regarding safety, upper GI events of nausea and vomiting comprised the majority of AEs. These events were predominantly mild and moderate in severity, dose-related and similar in frequency to those observed in prior trials of pemvidutide. Rates of lower GI AEs including diarrhea and constipation were notably low. This AE profile was observed in the absence of dose titration at the 1.2 mg and 1.8 mg doses and with a limited 4-week dose titration at the 2.4 mg dose. One subject (2.4%) experienced a serious adverse event of nausea and vomiting requiring rehydration at the 2.4 mg dose. Treatment discontinuation rates were 28.2% in subjects receiving placebo and 24.0% in subjects receiving pemvidutide. The majority of placebo discontinuations were due to withdrawal of consent, while approximately half of the withdrawals across the pemvidutide dose groups were attributed to GI AEs. These discontinuations occurred almost entirely in the first 16 weeks of treatment. The protocol did not allow for dose reduction due to intolerability as employed in other incretin trials.

https://finance.yahoo.com/news/altimmune-announces-positive-results-week-110000433.html

Echo Lake Capital Offers to Acquire Quince Therapeutics

 

  • Offers $1.60 per share in cash

  • Offer represents 90% premium to yesterday's closing price

  • Stock currently trades below its current cash and investments balance of $2.55 per share

  • Company also has other assets including earnouts and NOLs

  • Company is not developing or selling any drugs

  • Offer not contingent on outside financing

Scinexis: started at Buy by Guggenheim

 

  • Guggenheim has initiated coverage on SCYNEXIS Inc (NASDAQ: SCYX) with a Buy rating and a price target of $8.

  • The anti-infectives space remains out of favor with investors, but SCYNEXIS offers a unique opportunity given that ibrexafungerp is already approved to treat vulvovaginal candidiasis (VVC), and has shown promising early data against more severe invasive infections, writes the analyst.

  • In October, SCYNEXIS announced a new strategic direction to refocus its resources on the clinical development of ibrexafungerp for severe, hospital-based indications.

  • The company intended to out-license Brexafemme (ibrexafungerp tablets) VVC and sought a U.S. commercialization partner.

  • Guggenheim is optimistic about ibrexafungerp showing non-inferiority in the MARIO trial (data expected 1H 2024), establishing ibrexafungerp as the step-down option of choice for fluconazole-resistant invasive candidiasis.

  • Data from the ongoing FURI and CARES trials (data expected in 1H 2024) would allow for the expansion of ibrexafungerp into refractory fungal infections.

  • Successful development of an intravenous option is also of interest given the possibility of allowing ibrexafungerp to be used as initial IV therapy and then providing the patient with the same product for oral step-down.

  • Positive results would likely set the stage for ibrexafungerp's potential FDA approval, with market entry expected in late 2024/early 2025.

  • Guggenheim estimates peak ibrexafungerp sales of $185 million, driving a significant potential upside to SCYX shares from current levels.

Monday, March 20, 2023

Prestige Bio fast tracked for Treatment of Pancreatic Cancer

 Prestige Biopharma has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for PBP1510 (International Non-proprietary name: Ulenistamab), in the treatment of unresectable or metastatic pancreatic adenocarcinoma (PDAC) that has relapsed following and/or is refractory to at least one line of prior therapy.

PBP1510 targets Pancreatic Adenocarcinoma Upregulated Factor (PAUF), a tumour-specific protein, found to be overexpressed in majority of pancreatic cancer cases. PAUF overexpression promotes key cellular functions, including proliferation, migration, invasion, and growth of pancreatic cancer cells, and contributes to the development of acquired resistance to chemotherapeutic agents. PBP1510 is designed to target these key biological mechanisms that results in limited effectiveness of current treatment options and rapid progression of pancreatic cancer.

By effectively inhibiting the tumorigenic effects of PAUF overexpression in preclinical models, PBP1510 represents a promising therapeutic strategy for addressing the unmet medical needs of pancreatic cancer patients. A global Phase 1/2a clinical trial is currently underway in the United States, Europe, and Asia, with the aim of bringing this innovative therapy to the clinic.

The first-in-human Phase 1/2a study is an open-label, multicentre, two-part study in patients with advanced/metastatic pancreatic cancer. Phase 1 is a dose-escalation phase, wherein PBP1510 will be administered, either as monotherapy or in combination with gemcitabine, in two separate dose-escalation cohorts. From Phase 1 part of the study a recommended Phase 2a dose (RP2D) will be determined based on the analysis of pharmacokinetics, safety, and efficacy data. Phase 2 is a dose-expansion phase, wherein PBP1510 at the RP2D in combination with gemcitabine will be administered to evaluate efficacy and safety of PBP1510.

Overall, the Phase 1/2a study aims to collect important safety data on the use of PBP1510 as a monotherapy or in combination with gemcitabine and explore the efficacy of a combined PBP1510 and gemcitabine regimen. The study will substantiate the preclinical findings of PBP1510’s synergistic antitumour activity in combination therapy with gemcitabine without increased toxicity, as anticipated from their distinct mechanisms of action.

With Fast Track designation from the FDA, PBP1510 represents a promising advancement in the treatment of pancreatic cancer. Prestige Biopharma intends to take full advantage of the benefits offered by the designation to provide faster access for patients in need.

https://www.biospace.com/article/releases/prestige-biopharma-receives-fda-fast-track-designation-for-pbp1510-in-the-treatment-of-pancreatic-cancer/