Sarepta will finally reveal the high dose LGMD2E data from the gene therapy SRP-9003 on Monday morning and put this highly underappreciated program on the big podium. In a surprising Friday post-market closing press release, Sarepta announced that on Monday, June 8, 2020 at 8:30 am ET, it will host a webcast and conference call […]
Cardiol Therapeutics – Pharmaceutical Cannabidiol (CBD) Long-Term Value Creation Will Lead the Way to Acute Myocarditis Clinical Trial
Cardiol Therapeutics (CRTPF) is a Canadian company that completed its initial public offering (IPO) on the Toronto Stock Exchange (the “TSX”) back in December 2018 and trades mainly on the TSX under the symbol CRDL while in the U.S. it trades on the OTCQX under the symbol CRTPF. Cardiol is focused on producing pharmaceutical cannabidiol […]
CHFS – Can The COVID-19 Pandemic Become the Inflection Point?
A Reported Shortage of Dialysis Machines Puts a Focus on Aquadex. Aquadex Smartflow System is being used to remove excess fluid from patients being treated with fluid resuscitation as part of the treatment for COVID-19, and in some cases is helping to relieve the shortage of dialysis machines at a growing number of hospitals. A […]
February 3 Biotech Update
The market is off to a good start this morning but I suspect we fade. The old saying goes that markets never bottom on Fridays and typically when you have a big selloffs on Friday and a bounce Monday morning, the bounce ultimately fades and fails. We will see. We still have some macro that […]
The PTI Bear Case
Proteostasis Therapeutics is a small biotech company focused on the development of treatments for cystic fibrosis. To their credit, they have 3 compounds, each of a different class in the clinic. This is crucial because the current standard of care for CF relies on combination therapy. Vertex laid out a blueprint for CF treatment through […]
January 16 Addendum
The link I sent sends you to the twitch front page. You can go to videos to see the replay or this link below should send you there. https://www.twitch.tv/chimeraresearch/videos
Another Look at TPTX
With the entrance of RET inhibitor TPX-0046 into P1/2 last November, fast moving Turning Point Therapeutics now has a full slate of 3 clinical candidates. Soon, another ALK inhibitor is expected to join the fray. Pipeline: The pivotal repotrectinib studies in ROS1 and TRK are ongoing with interim results from some registrational cohorts expected during […]
All Eyes On SAGE Ahead of JPM
SAGE has been a very volatile stock even since its early days. Hopes have been dashed only to be renewed by another promising aspirant. Only weeks ago, the subject of this affection was SAGE-217. It traded as high as $198 in July before falling to $56 on December 5 when it was announced the drug […]
Is APLS a Buy Ahead of Data?
Apellis Pharmaceuticals is developing treatments through targeting the human complement cascade. Its most advanced product is C3 inhibitor APL-2. Pivotal trials are ongoing in PNH and geographic atrophy in age-related macular degeneration, or GA, where C3 is known to be a primary disease driver. Results from a highly anticipated head-to-head study of APL-2 vs eculizumab […]
The CNST Bull Case in a Single Slide
Constellation Pharmaceuticals presented some compelling data at ASH. In early results from 15 JAKi naive Myelofibrosis patients treated with a combination of BET inhibitor CPI-0610 and ruxolitinib, 12 (80%) achieved a Spleen Volume Response of at least 35% (SVR35). The numbers indicate 8/11 (73%) of the newly added patients had a response. This is a […]
Taking a BET on CNST
Constellation Pharmaceuticals is focused on the development of cancer therapeutics by targeting the epigenetic pathway. It has EZH2 and BET inhibitors in mid-stage clinical studies. Excitement in the stock is driven by the BET inhibitor CPI-0610. There is substantial interest in this class of compounds. While small biotechs often lead the way with novel targets, […]
Initiating: Turning Point Therapeutics
San Diego based Turning Point Therapeutics is an oncology focused biotech that completed a successful IPO in April this year, raising about $190M with pricing at $18/sh. As the stock continued to climb, TPTX quickly filed for a secondary on September 3rd, adding another $200M to its cash hoard. The company is now very well […]
ARQL- Quick Take
I have been following Arqule off and on for quite some time. The company has been reborn after so many lost years pursuing its failed MET inhibitor ARQ 197. Hopes are high for lead asset ARQ 531, a competitive inhibitor of BTK. Arqule also has a pipeline of 2 AKT inhibitors and an out-licensed FGFR […]
Zogenix Acquires Modis Therapeutics
Aug 27, 2019 by Jason Chew Zogenix announced yesterday that it is acquiring privately held Modis Therapeutics. It gains access to the late stage substrate enhancement therapy, MT1621, for the treatment of Thymidine Kinase 2 deficiency (TK2d). Zogenix is paying $250M upfront, consisting of $175M cash and $75M stock. Modis may also receive milestones of […]
Coverage Continuation- ARVN
Aug 23, by Jason Chew Arvinas is one of the newer names I’ve been covering. The company is developing a new class of drugs with a new modality known as Targeted Protein Degraders, coining the technology PROTAC (Proteolysis Targeting Chimera). PROTACs work in a completely different way from current small molecule drugs. Most of today’s […]
Coverage Continuation- ZGNX
Aug 19, 2019 by Jason Chew Zogenix ran into some unwelcome surprise this April when FDA kicked back its ZX008 (Fintepla) NDA with a Refuse to File letter. The two deficiencies cited were the lack of a non-clinical toxicology report and submission of an incorrect clinical dataset. The request for additional toxicology studies was unexpected; management believed […]
Coverage Continuation – ZYME
Aug 14, 2019 by Jason Chew Zymeworks is perhaps one of my favorite stocks. The small/mid cap Canadian biotech has expertise in bispecific antibody technology. Bispecifics are an exploding field, now with over 50 antibodies in the clinic. Their flexibility allows for a multitude of applications. Some analysts believe bispecifics will drive growth in the […]
Coverage Continuation: SAGE
It has been a long time since my last article, but with the forum down, there is a lot to catch up on. My coverage will focus on the eight stocks in my portfolio. These are: SAGE; ZYME; ZGNX; ARVN; AMRN; ARQL; CALA; AXSM. We’ll start with SAGE: Things continue to hum along for Sage […]
August 12th Biotech Update
Another week with another set of macro concerns. I was waiting to see if there would be any interesting biotech news but I guess not. There was some late last week but this morning seems driven by macro (your choice between Argentina and/or Hong Kong and/or China trade). I see little on the horizon that […]
DMD Data by Giant Pharma Pfizer Kept Sarepta In the Driver Seat with Superior Drug
Pfizer (PFE) presented its first set of data (n=6) from the mini-dystrophin gene therapy for DMD (PF-06939926) at the PPMD conference on June 28th. Much speculations surrounded Pfizer’s decision to report the dystrophin quantification using mass-spec. Sarepta (SRPT) has used WB-western blot since 2015 during the Eteplirsen FDA approval process. Sarepta has also used it in […]
