March 1, 2021

Impact: High

Reata has submitted an NDA to the FDA for bardoxolone methyl for the treatment of chronic kidney disease caused by Alport Syndrome. Reata has requested priority review for the application. The submission was based on the results of the phase 2/3 CARDINAL study which demonstrated statistically significant improvement in  kidney function as measured by eGFR with bardoxolone vs placebo. Read more

February 14, 2021

Impact: High

During its Q4 2021 earnings call, Prothena gave an update on its prasinezumab clinical program for Parkinson’s disease. During the clinical overview, Prothena highlighted that prasinezumab specifically targets the C-terminus of alpha-synuclein and is the “first antibody to show significant slowing of motor progression and improvements in imaging biomarkers.” The company stated that preclinical studies had shown that targeting the N-terminus was suboptimal and proposed it as a potential hypothesis on why Biogen’s cinpanemab failed, a point later reiterated during the Q&A.

Prothena remains very encouraged by efficacy signals observed in its Phase 2 PASEDENA Part 1 study. Data from pre-specified exploratory subgroup analysis from the PASEDENA Part 1 study will be presented at the ADPD conference in March. Prothena expects Roche to present results from PASEDENA Part 2 at a future medical conference.

Roche is expected to initiate the Phase 2b study of prasinezumab in Parkinson’s disease in Q2 2021 with details to come in Q2 as well. Prothena will earn $60M clinical milestone payment upon dosing of first patient. Read more

February 3, 2021

Impact: High

Both Pfizer and Amgen provided updates on its BCMA bispecific programs in Q4/FY 2020 earnings calls on February 2.

  • Pfizer’s BCMAxCD3 bispecific, elranatamab (PF-06863135), received FDA Fast Track designation in late January for the treatment of multiple myeloma patients who are refractory to at least one proteasome inhibitor, one immunomodulatory drug and one CD38 antibody.
    • The potential registration-enabling phase 2 trial has been initiated and the first patient is expected to be dosed this month.
    • In the Q&A, when asked about differentiation of elranatamab in the competitive landscape, Pfizer emphasized the 83% response rate in a heavily pretreated population with a significant number of complete responses and a nice tolerability profile with the opportunity for best-in class.
      • “So although it’s filled with several entrants, I think we have an opportunity to aim for being absolutely in the first wave here and with a really nice best-in-class profile. We’re moving with first opportunity we see for accelerated approval in triple refractory patients that either have seen no prior BCMA-based treatment or have seen prior BCMA treatments such as ADC or CAR-T. So we are planning such cohorts to start soon with a potential for registration. And we’re moving into second and third line, and other combinations that are used in order to come to first and second-line opportunity.”
  • The phase 1 trial of Amgen’s BCMAxCD3, pavurutamab (AMG 701), has been paused while Amgen discusses protocol modification to optimize safety monitoring and mitigation with FDA.
    • This is the first time Amgen has communicated on this hold, despite a ClinicalTrials.gov update on December 18 stating that the trial was not recruiting.
    • Currently enrolled patients demonstrating benefit may continue to receive treatment and enrollment is expected to resume in H1 2021.
    • When asked about safety issues in the Q&A, Amgen remains “bullish” on its BiTE program and stated that “cytokine release syndrome is clearly the single challenge that sits before the entire field.”

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January 15, 2021

Impact: Moderate

  • Ipsen will present 26 abstracts at TOXINS 2021.
  • A key presentation titled AbobotulinumtoxinA: Evidence for Long Duration of Response from 5 Patient Populations demonstrates long-duration of Dysport.

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November 19, 2020

Impact: High

  • Results for Affimed’s AFM13 (CD30/CD16A ICE; Innate Cell Engager) in combination with Keytruda (pembrolizumab) in a phase 1b study for r/r Hodgkin Lymphoma patients have been published in the journal Blood. Key efficacy data included (n=30):
    • ORR (Objective Response Rate): 88% (CR rate: 46%) at the recommended dose level
  • Investigators deemed the combination to be safe and well tolerated with adverse events that were manageable.

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November 10, 2020

Impact: Moderate

Oramed is planning two new phase 2 studies to evaluate the efficacy and safety of its oral insulin candidate, ORMD-0801 in T2DM patients with NASH. The studies are due to start in November 2020.

  • The first study is a double-blind study that will randomize 36 subjects in a 2:1 ratio to receive either 8 mg ORMD-0801 or placebo.
    • Patients will receive one capsule twice a day (once in the morning approximately 30 to 45 minutes prior to breakfast and no later than 10 AM, and once at night between 8 PM to Midnight and no sooner than 1 hour after dinner).
  • The second study is an open-label study to evaluate once-daily and twice daily regimens of ORMD-0801. Patients will receive either 8 mg QD or 8 mg BID.
  • Both studies are primarily safety studies that will evaluate preliminary efficacy as secondary outcome measures.
  • The studies are due to readout at the end of 2021.

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October 8, 2020

Impact: Moderate

  • Krystal Biotech announced the presentation of preclinical data supporting the ongoing development of KB301. Data will be presented at the American Society for Dermatologic Surgery (ASDS) 2020 Virtual Meeting to be held October 9-11, 2020.
  • KB301 is an investigational therapy designed to restore collagen homeostasis locally via directed expression of full-length human type III collagen gene (COL3A1).
  • Poster titled, In Vitro and In Vivo Pharmacology of KB301, an HSV-1-Based Gene Therapy, for the Treatment of Superficial Skin Depressions highlights that:
    • In vitro, KB301 readily transduced primary aged male and female human dermal fibroblasts (HDFs) and induced full-length COL3 expression, proper maturation, and subsequent secretion.
    • In vivo, successful vector transduction and subsequent COL3 expression was observed in a dose-dependent manner at both the transcript and protein levels in young and aged immunocompetent mice.
    • Safety looks promising as high concentrations of KB301 were non-cytotoxic to the cells in vitro.
  • A phase 1 trial, PEARL-1, is currently recruiting and expects to enroll 22 patients. The study will evaluate the safety and tolerability and initial efficacy of 2 different dose levels of KB301 injections in healthy buttock skin (NCT04540900).

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September 15, 2020

Impact: Moderate

At two recent investor conferences, Revance Therapeutics’ CEO, Mark Foley, provided updates on the company’s activities.

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September 21, 2020

Impact: High

inThought Research and Bloomberg Intelligence were joined by expert speakers in the field of Spinal Muscular Atrophy (SMA), Adrian Krainer, PhD, and Thomas Crawford, MD, to discuss the recent approval of the third disease-modifying therapy, Evrysdi. Key insights provided include:

  • Patients currently stable with Zolgensma or Spinraza will likely not be switched to Evrysdi. Due to a lack of long-term data, Dr. Crawford described a “wait and see” approach for the new drug.
  • The speakers questioned whether an oral drug will reduce drug adherence in patients treated with Evrysdi.
  • The main barrier to treatment with both Zolgensma and Spinraza is access. Evrysdi will be prescribed to patients without access to a hospital setting, not eligible for the one-time gene therapy Zolgensma, and patients with spinal deformities, making Spinraza’s intrathecal injections challenging.
  • There is a momentum with newborns being treated with Zolgensma, and parents are drawn to the safe one-time treatment. Although, there is a concern regarding Zolgensma’s safety in older patients (intrathecal injections).
  • Both speakers agreed that a medical device, such as an implantable drug pump for Spinraza treatment, would allow it to better compete with the new therapies.
  • All three treatments have comparable efficacy and safety; a biomarker, such as measuring neurofilament accumulation, should be implemented to accurately and sensitively measure outcomes of the different treatment options.
  • Additionally, the speakers find it doubtful the new oral drug will drive down the price of alternative therapies, as there is not a substantial price difference when a patient peaks the weight-adjusted pricing.

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September 10, 2020

Impact: Moderate

Gemini Therapeutics enrolls first patients for its phase 2a ReGAtta study to evaluate the safety and efficacy of GEM-103 for the treatment of patients with GA secondary to dry AMD. The study will recruit up to 80 participants and will include both patients from the phase 1 study as well as treatment-naïve patients. Participants will be administered with IVT injections of GEM-103 dosed either monthly or every other monthly. Read more