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 as a whole. I also discuss updates from CYCN and ADVM. Check out our sponsor at Gallant.com and use promo code: BIO to save when you enroll...

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 Follicular Lymphoma. We also talk about the recent positive data that was seen in the effects of Ublituximab in Relapsed Multiple Sclerosis....

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 Uniqure and Bluebird Bio. Sangamo also has a number Phase 1/2 programs that will have readouts in late 2021 as well as a large preclinical pipeline....

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 recently had negative data in a Phase 2 trial of Gout, but because upcoming catalysts are for other programs, I see an opportunity. I also discuss new...

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 the odds of getting through clinical trials successfully are extremely low. Here, I look at the existing data in $ATHA, $ANVS and $LGVN to see if...

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 subsector. 3:50 $CRTX 13:40 $AVXL 16:56 $ALEC 23:11 $CYCN Presentation slides:...

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 on #SAVA: https://youtu.be/j8sHp-M50NE https://youtu.be/kDuL2l7S2Dg Presentation slides:...

4D Molecular Therapeutics ($FDMT) has developed a new platform to create synthetic capsids that aim to improve gene therapy. They have candidates in Ophthalmology, Cardiology and Pulmonology that could rival existing technologies. In Ophthalmology, their R100 vector has shown minimal inflammation and neutralizing antibody generation in non human primates. Clinical data is expected in 2021, which could make or break the hype surrounding the company. Aurinia Pharmaceuticals ($AUPH) received FDA...

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 is also looking at treating Ewing Sarcoma with their ETS inhibitor. They only have early data, to date, but so far it is positive and justified...

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 characteristics that will define its efficacy and safety profile. In this video, I contrast the companies and discuss the data to date. If you want...

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 Therapeutics ($CRSP). To finish up the episode, I provide a follow up on a my discussion on Anavex ($AVXL) to discuss some peculiar points about...

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 I discuss the implications and future catalysts. Anavex's ANAVEX2-73 (Blarcamesine) is being investigated for Alzheimer's Disease, Parkinson's...

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 great detail along with a mock model. Biogen's Aducanumab was decidedly voted against in a recent FDA advisory committee with regards to the evidence...

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 the drug at. We also talk price targets and timing, which more often than not, is the most important factor when making a trade. Check out more...

Cyclerion effectively fails in their Phase 2 Sickle Cell Disease trial but shows mixed CNS data. I go through the positive aspects of the data and discuss the potential for the company (and my position). In the latter part of the video, I do a follow up to the Hepion interview I did with the CEO, Dr. Robert Foster. I also talk about how I plan to play the stock given the upcoming catalysts. I also touch on Amgen and their AMG510 Phase 2 data that was released in early Oct/2020. If you want to...

Hepion Pharmaceutical's (#HEPA) CRV431 is a broad cyclophilin inhibitor that has shown preclinical efficacy to reduce fibrosis in NASH. HEPA is about to release data from their Multiple Ascending Trial P1 study, which will be followed by data from their Phase 2a study later this year/early 2021. In this interview, I discuss the company's progress with Hepion CEO Robert Foster and where they might be headed in the future. If you want to help out the show (or join the discord), take a look at...

Mirati's KRAS G12C molecule MRTX849 has shown some efficacy in NSCLC, CRC and AC in a small patient population. At the end of Oct/2020, new data will come out that may rival Amgen's AMG510. Regeneron announced this week positive data in their Antibody Cocktail for the treatment of COVID-19. Patients that were seronegative had the most profound effect, likely due to the significant viral loads in these patients. In this video, I also touch on Exact Sciences and their update on liquid biopsy...

Cyclerion is switching gears from their failed trials in 2019 into Sickle Cell Disease and Neurologic Disease. By targeting soluble guanylate cyclase, they are able to promote cGMP and vasodilation. This has big potential for both indications, which we are expecting a readout in the next couple weeks. I also talk about updates from TRIL, SAVA, GILD/IMMU and ETNB. If you want to help the show, you can donate here: https://tips.pinecast.com/jar/breaking-biotech Follow me on twitter...

Evofem is preparing to launch their non-hormonal, on-demand female contraceptive, Phexxi. I talk about the company's expectations on market penetration and what might be causing the difference with regards to valuation. Odonate's Contessa trial showed an improvement in progression-free survival with their oral taxane therapy for breast cancer (Tesataxel), but the treatment also had significant side effects that might be a cause for concern. I also talk about the Complete Response Letters...

BioXcel (#BTAI) is a clinical-stage biotechnology company looking to commercialize neuroscience and oncology products developed with the help of their unique AI technology. I go through the data they have seen to date and upcoming catalysts that are likely to significantly affect the stock in the short term. As well, I touch on Cassava Sciences' update on PTI-125 and an interesting coincidence regarding the structure of the molecule. Credit goes to twitter user: @NoseRubInvest for finding...

AstraZeneca (#AZN) publishes their Phase 1/2 data of their SARS-CoV-2 vaccine candidate ChAdOx1 nCov-19. I go through the side effects, humoral efficacy and cellular response and contrast the results to Moderna's (#MRNA) candidate, MRNA-1273. I also talk about a few other biotech companies, notably: $TRIL, $VKTX and $BLUE. If you want to help the show, you can donate here: https://tips.pinecast.com/jar/breaking-biotech You can also open an account at Tastyworks using my referral code:...

In this episode I go through a few different biotech companies that I have touched on in the past (some which I have current positions). I close out the episode talking about Moderna and the publication of the interim data from their Phase 1 study for MRNA-1273, a vaccine for Sars-CoV 2. Time stamps: $RGNX 1:14 $ODT 7:09 $AXGT 9:20 $CBIO 12:44 $MRNA 18:07 If you want to help the show, you can donate here: https://tips.pinecast.com/jar/breaking-biotech You can also open an account at...

After delays in scheduling their advisory committee meeting, the FDA finally sends Intercept Pharma ($ICPT) a Complete Response Letter. In the letter, the FDA says that the benefits of Ocaliva shown via surrogate histopathologic endpoints do not sufficiently outweight the risks to support accelerated approval. Intercept will likely need to collect outcomes data to finally get FDA support but this will add significant time before approval. Meanwhile, NGM Biotherapeutics ($NGM) and Akero...

Actinium Pharma (#ATNM) is commercializing Antibody-Radiation Conjugates (ARC) for the treatment of diseases that require conditioning regimens (Bone Marrow Transplant, Stem Cell Transplant, Adoptive Cell Therapies). The current state of total body irradiation leaves patients with significant side effects and excludes a large demographic due to potential lethality of the conditioning treatment. ATNM is testing a targeted approach that could allow for substantially more patients to be treated...

Iovance (#IOVA) confirms a profound effect of LN-144 in metastatic melanoma with interim data from their 4th cohort. They also showed early data in NSCLC, which is an important indication for the company's future. Karyopharm's (#KPTI) Selinexor is approved for 5th line Multiple Myeloma and is currently being tested in other diseases. They have a number of upcoming catalysts that are likely to maintain the stocks value, and I discuss the details of my rough model for estimating value. Warning:...