Esperion – BA First Phase III Successful, But Stock Down on Safety Confusion & Some Efficacy Concerns
BioInvest BREAKING News – This morning, Esperion (ESPR) delivered positive Phase III safety data for bempedoic acid (BA). Despite delivering a highly significant 20% LDL reduction for those on treatment – expectations were for 20-30% LDL reduction based on several previous BA studies. The low-end for efficacy combined with safety concerns unrelated to treatment with the drug development candidate have driven the stock down by more than a third, and excessively in our view.
BioInvest News – Nektar (NKTR) Continues Charge Forward With ‘181 & ‘214 Leading The Way. On the recent Q4 conference call, NKTR provided updates for both ‘181 and ‘214. The company has had two positive pre-NDA meetings with the FDA over the last two months regarding ‘181. One very important aspect discussed with the agency is a better abuse scheduling for ‘181 given its non-addictive properties than a Schedule 2 which is currently used for addictive opioids.
BioInvest News – SGMO – Powerful Deal With GILD/KITE For ZFNs In Next Generation Off-The-Shelf CAR-Ts – Raising BUY to 30 & TARGET PRICE TO 40 – Despite being pre-clinical and early stage, GILD is paying $150 million upfront in cash and up to $3.1 billion in milestones plus tiered royalties on 10 SGMO-derived cancer drug candidates (or $300 million per product). Combining the KITE/GIlD expertise and infrastructure – with SGMO – will create a transformative leader in the field.
BioInvest News – Nektar (NKTR) NKTR has signed the largest single product deal in biotech history with BMS for their leading I/O development candidate NKTR-214. Clearly BMS paid handsomely for what may have been the most attractive asset in the space.
BioInvest News – Madrigal (MDGL) – MGL-3196 Delivers Positive Phase II HeFH Data – MDGL has released positive top-line HeFH Phase II data. The trial enrolled 116 patients who were not at their LDL-C goal and were randomized in a 2:1 ratio to receive either MGL-3196 or placebo, in addition to their current cholesterol lowering regimen, which included approximately 75% taking high intensity statins (20/40 mg rosuvastatin or 80 mg atorvastatin), and about 2/3 of patients also taking ezetimibe.
BioInvest News – NVAX – Delivers Some Positive Phase II Immunogenicity Data In COPD…Investors Wanted an RSV Update – NVAX reported top line data from the Phase II older adult safety and immunogenicity trial (E205) demonstrated the benefit of adjuvant formulations and two-dose regimens in patients with RSV and chronic obstructive pulmonary disease (COPD).
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.
The Medicines Company (MDCO) Inclisiran FDA Path Clear After End of Phase II Meeting – Lower Risk, Value Enhanced
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 Access to Management Webinar – Madrigal (MDGL)– We are pleased to announce another BioInvest “Access to Management Call.” Below is the link to the recorded webinar presentation with the Senior Management Team of Madrigal.
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 – 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 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 Update – The Medicines Company (MDCO) – Amgen’s FOURIER Outcomes Trial Successful – After the close, AMGN announced the long-awaited results of the outcomes trial with Repatha – its PCSK9 monoclonal antibody.
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 Access to Management Webinar – Anthera (ANTH)– We are pleased to announce another BioInvest “Access to Management Call.” Below is the link to the recorded webinar presentation with the Senior Management Team of Anthera.
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 Access to Management Webinar – Novavax (NVAX)– We are pleased to announce another BioInvest “Access to Management Call.” Below is the link to the recorded webinar presentation with the Senior Management Team of Novavax.
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…