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 […]
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 […]
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 […]
Opinion: No, The Antibiotics Market Is Not Broken
In light of slow drug launches and disappointing sales, there is a growing chorus of voices saying the antibiotics drug market is broken; that the government must step in to save one of our most important resources. With no profits to pursue, it is said, companies will abandon antibiotics, leaving society vulnerable to the spread […]
Celldex: Quick Take
Celldex has struggled for a couple years now since its brain cancer drug Rintega failed in 2016. In Q2 this year, results from a pivotal study of the gpNMB antibody drug conjugate, Glembatumumab Vedotin, may change the company’s fate. Glembatumumab is being tested in women with triple negative breast cancer with >25% gpNMB expression. Patients […]
Achaogen: Is It A Buy?
South San Francisco based Achaogen is on the verge of gaining FDA approval for its bacteria fighting drug Plazomicin. The decision date has been set for June 25 with an Advisory Committee (AdCom) meeting likely to occur in the April-May timeframe. If the rulings are positive, the drug will be launched this year. Plazomicin is […]
AGIO- Pivot to PKD
Agios Pharmaceuticals came to market in 2013 as a promising cancer metabolism company. Three years later, it has pivoted to rare disease. AG-120, an IDH1 mutant inhibitor being developed for oncology indications, remains the most advanced pipeline compound, but is no longer the main value driver. For that we look to AG-348 and AG-519, mid […]
ASH Update: TGTX
ASH 2016 has turned out to be an important convention for many small biotechs, and will likely be remembered as a break-out event for CAR-T therapies. Results from the Phase 1 TGR-1202/Ibrutinib combination study may prove crucial for the future of TG Therapeutics. Escalating doses of TGR-1202 ranged from 400mg to 800mg with a corresponding 420mg of […]
ASH Update 2: BLCM
Bellicum Pharmaceuticals’ leading clinical candidate is BPX-501, individualized T-cells transduced with a controllable suicide gene. They are being studied for use as an add-back following T-cell depletion during Haploidentical Stem Cell Transplant (HSCT). For individuals with certain hematological disorders, whether malignant or not, stem cell transplants represent the only hope for a cure. Siblings make […]
ASH Update: TRIL
Trillium Therapeutics presented interim data from its ongoing Phase 1 trial of the SIRPaFc TTI-621 at ASH this past weekend. These highly anticipated results gave a deeper look into the molecule’s safety and efficacy profile as well as potential mechanism of action. Trillium is at the forefront of a scramble to target the SIRPa-CD47 checkpoint […]
BLCM: Staying on the Sidelines
Bellicum is a small biotech developing drugs in the hot field of adoptive cell therapeutics. Their lead candidate is a personalized T-cell product for use after stem cell transplants to prevent early infections. These cells are designed with a safety switch as a safeguard. The early stage pipeline consists of CAR-T and TCR based immunotherapies. BPX-501: To prepare […]
Achillion’s Pivot To Orphan Diseases
Through many years, Achillion focused on HCV, among the biggest prizes in drug discovery. The problem is they entered the field late while working on a very popular target. A nearly one year clinical hold on their protease inhibitor sovaprevir added to their woes. As work continued on HCV, by 2014, Achillion began a parallel development […]
