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.

Read more

Friday, September 18, 2020 11:00 AM EST

Bloomberg Intelligence and inThought host a call with Spinal Muscular Atrophy experts for an in-depth review of the spinal muscular atrophy (SMA) treatment landscape, which now includes approved drugs from Biogen, Novartis and Roche.

Read more

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

September 1, 2020

Impact: Moderate

  • Gilead has reached an exclusive licensing agreement for Jounce Therapeutic’s JTX-1811. JTX-1811 is a monoclonal antibody (mAb) that targets CCR8 to deplete immunosuppressive tumor-infiltrating T regulatory cells (Tregs). CCR8 is expressed by these Tregs and they are depleted by JTX-1811 treatment.
  • JTX-1811 has yet to enter the clinic, an IND is anticipated to be filed in the 1H2021.
  • Per terms of the agreement, Gilead will make an upfront payment of $85 million in addition to a $35 million equity investment ($120M total). Future milestone payments (clinical, regulatory, and/or commercial) have the potential to reach $685 million.
    • Jounce is also eligible to receive royalties ranging from the high single digit to mid-teens for worldwide sales.
  • Jounce will lead development of JTX-1811 through IND clearance, and after Gilead will have the sole right to develop JTX-1811.

Read more

inDemic provides curated research and science-based analyses.

Register at no cost to use the system and receive alerts at

September 3, 2020, Houston, TX – inDemic announces the launch of its COVID-19 knowledge management system. inDemic is a non-profit organization that provides information and synthesis related to a wide range of pandemic solutions to help medical researchers, policymakers, and the public make informed decisions. Read more

August 25, 2020

Impact: Moderate


Immunovant reported topline results for its Phase 2a ASCEND-MG trial of IMVT-1401, an SC anti-FcRN, in MG:

  • Three arms were included (6 weeks treatment period with weekly dosing): 340 mg IMVT-1401 weekly (N=5), 680 mg IMVT-1401 weekly (N=5), and placebo (N=5). Targeted enrollment rate was 21 patients, but trial was unblinded at 15 patients to accelerate phase 3 program. Patients are currently completing the OLE. Full results will be shared at a future medical meeting.
  • IMVT-1401-treated patients (N=10, pooled analysis) showed a mean 3.8-point improvement on the MG-ADL scale vs. a mean decline of +0.6 for placebo (p=0.029).
    • MG-ADL responder rates (>2 point improvement) were 60% for IMVT-1401-treated patients vs. 20% for placebo.
    • MG-ADL deep responder rates (>6 point improvement) were 40% for IMVT-1401-treated patients vs. 0% for placebo.
  • IMVT-1401-treated patients also showed a highly significant improvement on the MGC scale (composite of patient and physician assessments), with an average improvement of 8.0 points vs. a mean decline of +1.4 for placebo (p = 0.006).
    • MGC deep responder rates (>10 point improvement) were 40% for IMVT-1401-treated patients vs. 0% for placebo.
  • Strong IgG reduction: 59% for 340mg dose and 76% for 680mg dose.
  • Good safety profile, with no SAE or AE and no imbalance in headaches. Reduction in albumin were consistent with prior studies.

Immunovant will proceed with a Phase 3 trial in H1 2021.

Three new indications are expected to be announced over the next 12 months.

Read more