Newsletter October 2018
Notable Bioeconomy News
1. New Biotech startups overview
A. Biotech vets launch a neuro startup with $54.5M to kickstart R&D
B. LogicBio Prices IPO Below Range, Raising $70M to Test Gene Therapy
C. With $90M Round, Stoke Eyes a “Spinraza For Epilepsy” And a 2019 IPO
D. China's Innovent prices HK IPO near top end, raises $421 million: sources
E. 89Bio Joins NASH Chase with a Teva Drug and $60M in Funding
2. High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
New research suggests a large majority of people could already be primed to disable a key component of CRISPR-Cas9 gene editing therapeutics. In the study, blood samples from 48 healthy volunteers were exposed to Cas9 derived from Streptococcus pyogenes, one of the most commonly used in CRISPR R&D, 96% of people had T cell immunity and 85% had antibodies against the Cas9. Although immunogenicity has been a problem in the field of gene therapy for a long time, these are concerning results for CRISPR drug developers heading into clinical trials.
3. Free of Baggage, Dicerna Cuts Another RNAi Deal and Gets $200M
Interest has been rekindled in RNA interference medicines. Eli Lilly has teamed up with Dicerna in a $200M dollar deal to pursue RNAi medicines for pain, and cardiometabolic and neurodegenerative diseases. This deal was sparked after Alnylam received FDA approval for the first ever RNAi medicine in August. This deal marks another in a string of recent deals for RNAi medicines; promising leads for a field many companies have abandoned.
4. Fresh Data Add More Confusion to Eisai-Biogen Alzheimer’s Drug
In July, results from a Phase 2 study of BAN2401, the Eisai-Biogen Alzheimer’s drug, showed that the drug not only cleared plaques of amyloid protein, but also helped patients function better. However, there is controversy surrounding these results. Some of the patients in the study had the ApoE4 genetic mutation, putting them at higher risk for AD and potentially skewing results. Although there are caveats, Eisai and Biogen are continuing to move forward with BAN2401, hoping a larger Phase 3 study will clarify results.
Recently Pfizer and Eli Lilly released encouraging results from an early Phase 3 study evaluating the efficacy and safety of subcutaneous administration of tanezumab, a humanized monoclonal antibody for osteoarthritis (OA). The drug reduced pain in patients with knee or hip problems, marking success for an investigational class of pain medications known as nerve growth factor inhibitors. These results are promising in the race for the first FDA approval of a non-opioid treatment for osteoarthritis pain.
6. TherapeuticsMD wins FDA nod for hormone therapy, capping off drugmaker’s prolific year
TerapeuticsMD has received their third FDA approval this year with a hormone therapy for hot flashes associated with menopause. The drug, Bijuva, is an oral treatment that contains bio-identical versions of estradiol and progesterone. Coming into market next year, Bijuva could be a blockbuster drug, aiding the 80% of women that experience hot flashes during menopause.
XtalPi, an AI and computational biotech startup, has announced a Series B-1 extension with $46M. This new investment, led by China Life Healthcare Fund, brings XtalPi’s total funds raised to $66M. The majority of this money will go towards developing AI applications for R&D drug discovery and development. Ultimately, XtalPi hopes to connect global pharmaceutical companies with AI research tools to aid in drug research.
Therapeutic Research
1. Nature Chemical Biology: Identification of a cellularly active SIRT6 allosteric activator
2. Cell: Targeting Processive Transcription Elongation via SEC Disruption for MYC-Induced Cancer Therapy
3. Nature Medicine: Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent
4. Nature Medicine: Route of immunization defines multiple mechanisms of vaccine-mediated protection against SIV