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Absci is a synthetic biology company focused on developing better, faster paths to new medicines. Starting with their bioengineered E. Coli strain named SoluPro, Absci is able to manufacture complex biologics in a prokaryotic system, something that had never been done before. More recently, they have made acquisitions that have added on drug discov…
 
REGENXBIO and Abbvie announce a collaboration to co-develop RGX-314 for the treatment of wet AMD, diabetic retinopathy and other chronic retinal diseases. This massive announcement puts a lot of resources behind the commercialization efforts of RGX-314 in anticipation of an eventual approval. In the short-term, $RGNX benefits from a large upfront p…
 
Trillium Therapeutics is acquired by Pfizer for $2.26B! Finally, the small-mid cap biotech sectors sees good news with M&A activity that rippled through the sector. In this video, I talk about a few other companies that have CD47 assets that should see tailwinds from the TRIL acquisition. I also talk about the recent controversy surrounding Cassava…
 
Tg Therapeutics is a B-cell focused company that recently provided a Q2-2021 update of their commercial and clinical programs. In this episode, I talk with the CEO of the company, Michael Weiss, about some of the news they shared and what we can look forward to from the company. Help out the show (or join the discord) by becoming a patron at: https…
 
As Q2-2021 earnings season comes to a close, I decided to do a recap on multiple biotech companies I'm interested in, providing updates from each one's Q2 report. Commercial-stage biotech continues to be a risky place to invest, but there are some bright spots we can look towards that I'll share in this episode. Here is the breakdown: $BIIB - 00:45…
 
Geographic Atrophy is a progressive disease that leads to degeneration of the macula of the eye. Many companies have tried to develop therapies given the large market opportunity, but have been met with failure. This has not stopped new arrivals from trying their hand at improving patient outcomes. In this episode, I focus on the following companie…
 
Checkpoint Therapeutics ($CKPT) is an clinical stage oncology company that is focusing on developing treatments for solid tumors. Their lead candidate, Cosibelimab, is an anti-PD-L1 that has properties, which give it potentially more activity than checkpoint inhibitors that are currently on the market. In Q4-2021, they have an important pivotal rea…
 
Various companies in the Ophthalmology gene therapy space saw upsets from either an efficacy, safety or collaboration standpoint. However, three related companies have upcoming catalysts in the second half of 2021 that could turn sentiment around. They are: 4D Molecular Therapeutics, REGENXBIO and Clearside Biomedical. In this episode, I talk about…
 
Biogen's Aduhelm (Aducanumab) is approved under accelerated approval by the FDA for patients with Alzheimer's Disease. This shatters the bear narrative surrounding Biogen and signals a bullish turn for other CNS companies. However, the FDA is in a difficult spot given they will be forced to make more decisions about CNS assets that show no clinical…
 
Annovis' stock price recently moved higher after announcing Alzhiemer's Disease results from their ANVS401 compound. Despite the small sample size and statistical insignificance (p=0.13), there is tremendous excitement for the company's potential. Here, I compare Annovis' results to Cassava and Biogen and look at other upcoming CNS readouts to see …
 
Curis' CA-4948 is a first-in-class IRAK4 inhibitor that is in clinical development for AML/MDS and NHL. It's estimated that over 50% of AML patients have the IRAK4-L isoform, providing a nice patient population that could be eligible for this therapy. The company provided a recent update in anticipation of the European Hematology Association meetin…
 
Trillium Therapeutics held its R&D day on April 28th, 2021. There, they presented data updates on the effects of TTI-621 and TT-622 in lymphoma indications. They also announced 7 hematologic and solid tumor indications that they are going to focus on for the next year or so. In this episode, I go through these details and talk about the CD47 space …
 
TG Therapeutics is a B-cell focused company that has recently seen massive successes with their two lead compounds Umbralisib (UKONIQ) and Ublituximab. In this episode, I'm pleased to welcome CEO of TG Therapeutics, Michael Weiss. Michael and I discuss the company's commercial strategy since the FDA approval of UKONIQ in Marginal Zone Lymphoma and …
 
Sangamo Therapeutics is clinical-stage biotech company looking to commercialize a number of assets for the treatment of disease. Their furthest along program is a gene therapy treatment for Hemophilia A. The sector has seen some recent upsets with the FDA issuing a CRL to Biomarin for their HemA gene therapy, as well as some safety concerns with Un…
 
Selecta Biosciences is developing a platform to promote immuno-tolerance. Many treatment regiments have unwanted immune responses that can reduce the therapy's efficacy or cause immunity against the vector in a gene therapy. They are also seeking indications in autoimmune diseases. If successful, SELB's platform would see massive adoption. They rec…
 
Episode Notes In this episode, I do a follow-up to episode 084 looking at a few more biotech companies in the Neurodegenerative disease subsector. Alzheimer's and Parkinson's disease have massive patient populations that could take these companies to multi-billion dollar valuations. Unfortunately, these diseases are extremely difficult to treat and…
 
Episode Notes Companies with clinical assets for the treatment of neurodegenerative diseases are extremely high risk investments. With that risk comes the potential for a massive total addressable market, specifically in Alzheimer's and Parkinson's Disease. In this video, I discuss a variety of companies in the space and how I like to navigate the …
 
Cassava Sciences is commercializing Simufilam for the treatment of Alzheimer's Disease. They announced interim data from their open label study showing an improvement in ADAS-Cog11 and NPI, which led to a massive increase in the stock. In this video, I go over the data, how it compares to other trials and what's next. Check out my previous videos o…
 
4D Molecular Therapeutics ($FDMT) has developed a new platform to create synthetic capsids that aim to improve gene therapy. They have candidates in Ophthalmology, Cardiology and Pulmonology that could rival existing technologies. In Ophthalmology, their R100 vector has shown minimal inflammation and neutralizing antibody generation in non human pr…
 
Oncternal Therpautics ($ONCT) is commercializing a monoclonal antibody to antagonist the ROR1, a receptor upregulated in cancer. The data presented to date shows a powerful effect in combination with Ibrutinib in Mantle Cell Lymphoma, an aggressive form of Non-Hodgkin Lymphoma. They are also conducting studies in CLL and Breast Cancer. The company …
 
CD47 is the latest immuno-oncology target and multiple companies are commercializing molecules in blood and solid tumors. Two exciting companies in the space are Trillium Therapeutics and ALX Oncology. Both of them have molecules that target CD47 to stop the "don't eat me" response from macrophages. However, each therapeutic has some interesting ch…
 
The 62nd American Society of Hematology conference took place from December 5-8, 2020 where a number of companies presented compelling data updates. I give some background on Non-Hodgkin's Lymphoma then discuss updates from Trillium Therapeutics ($TRIL), Tg Therapeutics ($TGTX), Actinium Pharmaceuticals ($ATNM), Fate Therapeutics ($FATE) and Crispr…
 
Rhythm Pharmaceuticals gets FDA approval for Setmelanotide (IMCIVREE) for Chronic Weight Management in Patients with Obesity due to POMC, PCSK1 or LEPR Deficiency (Age 6 and older). The management team also gave pricing information, indicating $330/mg and blended pricing estimate of $290-300K/patient/year. The stock rose about 21% on this news and …
 
Episode Notes Rhythm Pharmaceuticals is tackling rare genetic obesity disorders with their MC4R agonist, Setmelanotide. They have 3 upcoming catalysts, the most important being the effect of the compound on a basket of high impact "loss of function" patients (POMC/LEPR-deficient heterozygotes, in particular). I go through all their catalysts in gre…
 
Krystal Biotech (#KRYS) is using a novel gene therapy platform to treat various rare diseases. Their primary indication is Dystrophic Epidermolysis Bullosa (DEB) with an upcoming Phase 3 readout in 2021. Michael McGuire from WX Capital and I discuss the bull case for rare disease indications, the epidemiology of DEB and what the company might price…
 
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