BioInvest News – IONS – Reports Positive TTR Data, Safety/Patient Death Causes Concern – IONS reported positive Phase III data for inotersen (TTR-Rx) NEURO-TTR results in familial amyloid polyneuropathy (FAP) patients showing a statistically significant benefit in the two co-primary endpoints of modified Neuropathy Impairment Score (mNIS+7, p<0.0001) and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN, P=0.0006) post 15 months of treatment.
Breaking News – Sangamo Therapeutics (SGMO) – PFE Global Deal for Hemophilia A Has Many Positive Implications – In a very competitive bidding process SGMO has inked a significant global partnership with Pfizer, who brings a wealth of value to SGMO, and notably validates the latter company’s gene therapy platform.
BIOINVEST BREAKING NEWS – The Medicines Company (MDCO) – Inclisiran FDA Path Clear After End of Phase II Meeting – Lower Risk, Value Enhanced – The Medicines Company announced that the primary endpoint of the Phase III registrational trial will be LDL-C levels. This is a major positive for MDCO – significantly reduces the risk and increases the value of Inclisiran.
BIOINVEST BREAKING NEWS – Esperion Therapeutics (ESPR) – FDA Shows Esperion “The Way to Approval” – Raising BUY to 40 and TARGET PRICE to 60
This morning, ESPR announced that the FDA has confirmed that the bempedoic acid Phase III development program is adequate to support an approval prior to their CVOT results, lifting one of the largest overhangs on the stock, and creating a clear path to an earlier-than-expected approval.
BIOINVEST BREAKING NEWS – Nektar (NKTR) – NKTR-181 Successful In Phase III SUMMIT-07 Trial & Sets The Stage For A Revolution In Pain Drugs – Raising BUY and TARGET PRICE
The SUMMIT-07 trial met its primary endpoint demonstrating highly significant improved chronic back pain relief with NKTR-181 compared to placebo (p=0.0019). Moreover, the safety and tolerability of ‘181 in SUMMIT supports its breakthrough profile for moderate-to-severe pain relief without the abuse and addiction that has caused the opioid epidemic of today
BIOINVEST BREAKING NEWS – The Medicines Company (MDCO) – FOURIER IS ACTUALLY MORE POSITIVE FOR INCLISIRAN THAN BEFORE – Being the first to market is sometimes a double edged sword and is certainly the case with the Amgen’s Repatha (and REGN/SNY’s Praluent). We were at the ACC where the FOURIER* data was presented on Friday.
BIOINVEST Update – The Medicines Company (MDCO) – – Discontinues ‘216 – AHA Meeting Next Week
News – MDCO Stops MDCO-216 After Pilot Trial Unsuccessful – The pilot trial did not show efficacy – there was no positive effect on atherosclerosis. Safety was fine. The data calls into question the viability of APO-A1 Milano as a target and the positive implications of HDL as well.
BIOINVEST Update – (New Recommendation) Madrigal Pharmaceuticals (MDGL) – Paul Friedman Is Back
NASH is a hot topic in Biotech since September when Allergan bought Tobira Therapeutics (TBRA) for a stunning $1.7 billion for a Phase II asset. The Tobira deal was done at a tremendous 500% premium and topped the $1.2 billion Gilead paid for a NASH drug candidate being developed by Nimbus Therapeutics.
BIOINVEST BREAKING NEWS – Special Update – Ziopharm (ZIOP) – 3 Patients Die In The IL-12 Trial. ZIOP is down sharply this morning (7/15) on the report that three patients have died in their ongoing multicenter Phase I trial of Ad-RTS-hIL-12 + orally administered veledimex in recurrent or progressive glioblastoma (GBM) or grade III malignant glioma.
BIOINVEST BREAKING NEWS – Special Update – ZIOP – Shows Rather Impressive Preclinical Data at ASGCT, Up Next Human Data at ASCO.
ZIOP was front and center at the 2016 Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Washington D.C. last week. The company presented exciting preclinical data for IL-12 in combo with a PD-1 that showed 100% survival. The non-viral Sleeping Beauty (SB) system was also on display. Read more…