MeiraGTx's gene therapy shows sustained benefit in inherited retinal disorder

• MeiraGTx (MGTX +2.5%) has announced 12-month data from the ongoing Phase 1/2 trial of AAV-RPGR, an investigational gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP). Data were presented at the American Academy of Ophthalmology.
• The primary endpoint of the trial is safety, with secondary endpoints assessing changes in visual function at pre-specified timepoints post-treatment.
• In the dose escalation phase of the trial, data at the 12-month time point demonstrated statistically significant improvement in retinal sensitivity in treated eyes in both the low (n=3) and intermediate (n=4) dose cohorts, with six of seven patients demonstrating improved or stable vision in the treated eye one year after treatment.
• Efficacy signals were observed at the first post-treatment assessments at three months, with improvements sustained or increased at 12 months.
• AAV-RPGR is well-tolerated till data, with no dose-limiting events occurred.
• MeiraGTx and development partner Janssen are preparing to initiate the pivotal Phase 3 Lumeos clinical trial of AAV-RPGR for the treatment of patients with XLRP.
• XLRP is the most severe form of retinitis pigmentosa, a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. The most frequent cause of XLRP is disease-causing variants in the RPGR gene.
• AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function.
• https://seekingalpha.com/news/3636738-meiragtxs-gene-therapy-shows-sustained-benefit-in-inherited-retinal-disorder
  

Dicerna/Roche's hep B candidate shows durable effect in early-stage study

• Dicerna Pharmaceuticals (DRNA -2.8%) has announced additional data from Phase 1 trial evaluating GalXC RNAi therapeutic RG6346, under development in collaboration with Roche for the treatment of chronic hepatitis B virus (HBV) infection. Data will be presented at the American Association for the Study of Liver Diseases.
• The results expand upon the interim results presented in August and demonstrate that four monthly doses of RG6346 treatment resulted in substantial and durable reductions in biomarkers of HBV disease activity as measured by reductions in hepatitis B surface antigen ((HBsAg)) levels lasting up to one year following the last dose. RG6346 was also shown to have a favorable safety and tolerability profile.
• 92% (11 of of the patients treated with RG6346 added to nucleos(t)ide antiviral therapy (Group C), had mean HBsAg reductions from baseline greater than 10 IU/mL by Day 112 (one month after last dose). 7 of 12 participants also achieved HBsAg levels below 100 IU/mL level that is associated with a reduced risk of progression to cirrhosis and hepatocellular carcinoma.
• After 448 days, the durability of HBsAg reductions was observed, the mean reduction in HBsAg from baseline was 25 IU/mL; one of these participants maintained  over 100 IU/mL reduction in HBsAg level from baseline at Day 448.
• https://seekingalpha.com/news/3636676-dicerna-roches-hepatitis-b-candidate-shows-durable-effect-in-early-stage-study
  

Vaccines set stage for fastest earnings rebound since ‘80s - DataTrek

• Earnings are expected to top 2019’s peak next year, a blistering pace for a recovery, and any upside to those expectations would make recovery valuations look better, DataTrek Research says.
• Wall Street consensus is for S&P 500 (SP500) (NYSEARCA:SPY) earnings of $168.38/share, 3.9% up from 2019’s $163.02/share, a recovery that would usually take about 3.5 to 4 years.
• But with strong Q3 results that annualize to $155/share, an effective vaccine, low rates and relatively good conditions for consumer spending, the quick turnaround looks doable, DataTrek says.
• Every sector in the S&P, with the exception of Energy, which won’t seen a return to profitability, is expected to post earnings growth of at least 5%.
• https://seekingalpha.com/news/3636739-vaccines-set-stage-for-fastest-earnings-rebound-since-80s-datatrek
  

Much of California ordered back into lockdown by Governor

• Governor Gavin Newsom has pulled "an emergency brake" on the state's reopening, with his actions just announced putting 94% of California's residents in the purple, or most restrictive tier.
• For comparison, 41 of the state's 58 counties are now in that purple tier vs. 13 last week.
• In his address (happening right now), Newsom points to increasing Covid-19 infection rates across every demographic and age group. He's also strengthening his mask mandate, ordering them to be worn statewide when outside the home.
• Now more streaming service than parks operator, Disney (NYSE:DIS) continues at the day's high, up 4.35%.
• https://seekingalpha.com/news/3636741-much-of-california-ordered-back-lockdown-governor-newsom
  

SQZ to Present Preclinical Data for Cellular Vaccine Platforms

SQZ Biotechnologies (NYSE: SQZ), a cell therapy company developing novel treatments for multiple therapeutic areas, today announced the presentation of first-time preclinical data for SQZ Antigen Presenting Cells (APCs) in combination with immune-oncology compounds in a poster at the 35th Annual Meeting of The Society for Immunotherapy of Cancer (SITC 2020) virtual poster sessions. A trial-in-progress poster for SQZ-PBMC-HPV-101 and additional posters of preclinical data from two proprietary cell therapy platforms, SQZ APCs and SQZ Activating Antigen Carriers (AACs), will also be presented. The presented data for both SQZ APCs and SQZ AACs summarize the robust CD8 T cell activation observed in the pre-clinical studies, supporting their potential as promising cellular vaccine platforms.

SQZ Biotech is a clinical-stage biotechnology company developing transformative cell therapies for patients with cancer, infectious diseases, and other serious conditions. Using its proprietary technology, SQZ has the unique ability to deliver multiple materials into many patient cell types.

https://finance.yahoo.com/news/sqz-biotech-presents-preclinical-data-114500154.html

CryoPort falls as Moderna's vaccine does not need deep freeze

• CryoPort (NASDAQ:CYRX) plunges as much as 18% after Moderna said its experimental COVID-19 vaccine would not need ultra-cold temperatures to be preserved.
• Moderna says new data showed its vaccine is stable at refrigerator temperatures for 30 days; by comparison, the vaccine being developed by Pfizer and BioNTech needs to be kept in deep freeze until a few days before use.
• https://seekingalpha.com/news/3636659-cryoportminus-13-after-modernas-vaccine-not-need-deep-freeze
  

Broad vaccine access expected in mid-2021: B of A

• Bank of America weighs in on the timeline for vaccine distribution after both Moderna (MRNA +8.0%) and Pfizer (PFE -3.8%) reported strong efficacy rates with safety levels anticipated by physicians to be acceptable when the final readouts come through.
• The firm says emergency use authorization designations are expected in the coming weeks with approval potentially coming before the end of the year.
• "Full BLA approval will likely require substantially longer follow up, including 6 months of safety follow up, although this could be shortened depending on perceived benefit/risk. This would put BLA filing on track for 2Q21, with full approval potentially coming shortly thereafter, in-line with our current view of broad access in mid-2021. On distribution, mRNA-1273 should prove easier to transport and administer than BNT162b2, requiring only 4 to -20C storage versus -70C storage in case of Pfizer/BioNTech's vaccine."
• The firm notes that Moderna's mRNA-1273 should prove easier to transport and administer than BNT162b2 due to the differences in storage temperatures.
• Early vaccine distribution to high-risk groups and frontline workers is anticipated well before broad distribution.
• https://seekingalpha.com/news/3636672-broad-vaccine-access-expected-in-middle-part-of-2021