Video Sponsor - http://workinbiotech.com/ In this episode, I go through a company called Vaxxinity, who are trying to develop vaccines for the treatment of various different diseases. They have a novel vaccine platform whereby they hope to induce both a humoral and T-cell response to specific proteins in various indications. One of their near-term readouts is a Phase 1 Part 2 data release from their UB-312 program in Parkinson Disease. This treatment is able to induce anti-α-synuclein...

In this episode, I take a look at 6 different biotechs and talk about recent updates in each one. Here is the breakdown: BTAI - 1:00 TGTX - 5:25 CRIS - 18:00 CKPT - 22:39 KPTI/SRRA - 31:07 Thanks to InfoPathways for being a sponsor of the show! Check them out for all your biotech IT needs at infopathways.com or call 410-751-9929. Help out the show (or join the discord) by becoming a patron at: https://www.patreon.com/breakingbiotech Follow me on twitter @matthewlepoire Send me an email...

Achieve Life Sciences is developing Cytisinicline for the treatment of smoking cessation. The drug has been available in Eastern Europe for years, but Achieve has taken the lead to bring this drug to market in developed countries. The safety profile is significantly better than both Chantix and Zyban and they are about to release data from the first of two Phase 3 clinical trials (ORCA-2 and ORCA-3). Given that there are 31M Americans who smoke and that e-cigarette usage is on the rise, there...

Kodiac Sciences is developing KSI-301 for a number of proliferative endothelial retinal disorders. Their recent P3 wet AMD data readout failed to show non-inferiority to Q8W dosed Eylea, a major upset for the company. However, KOD has a number of other upcoming P3 trial readouts that will be massive movers for the stock. In this episode, I go over the P3 data and talk about whether or not this is a buying opportunity. I also talk about Karyopharm and their recent regulatory update about the...

Karyopharm is an oncology company that is commercializing XPOVIO (Selinexor) in Multiple Myeloma and DLBCL. Selinexor blocks nuclear export thereby hindering the ability of cancer cells to block tumor suppressor protein activity. In this episode, I break down the bear and bull narratives of Karyopharm and discuss how the company has transitioned since entry of the new CEO Richard Paulson. Thanks to InfoPathways for being a sponsor of the show! Check them out for all your biotech IT needs at...

The NASH space has seen some setbacks in the last couple years, but a few players continue to make nice progress on their clinical trials. Madrigal Pharma announced positive topline data in their MAESTRO-NAFLD trial, which is an encouraging sign for their upcoming readout in NASH in Q3-2022. I discuss other players in the space including: ETNB, AKRO, VKTX, ICPT, IVA and NVO. Checkpoint Therapeutics announces positive topline data from their trial of Cosibelimab in metastatic cutaneous...

CymaBay Therapeutics is developing Seladelpar for the treatment of Primary Biliary Cholangitis (PBC). PBC currently affects around 130K patients in the USA and current available treatments do not address patient needs sufficiently. Seladelpar is an oral, selective PPARδ agonist that has been shown to regulate critical metabolic and liver disease pathways. CymaBay is currently enrolling for a registrational Phase 3 program in PBC with topline data expected in 2023. Data from prior trials show...

CRISPR is an exciting space, with a number of companies finally sharing early clinical data in 2021. NTLA benefitted the most from this by doubling in valuation after data release, while the other CRISPR companies continue to trend lower in the XBI bear market. Caribou Biosciences is one of the smaller EV CRISPR companies with their first clinical readout coming up in 2022. In this episode, I go through a short primer on all the CRISPR companies and discuss why I think Caribou will have a...

ALXO announces new HR-MDS data that disappoints! I talk about the comparison to Magrolimab data and why I think ALXO is still a buy. LGVN pushes data updates but is still a risk for a short squeeze! I talk about some of the stock statistics and fundaments to determine whether I'm going to place that short trade. The neurodegeneration space had an exciting 2021 and I provide an update on all the companies I covered earlier in the year. 2022 might prove to be more exciting with some big...

Curis is an oncology company trying to develop an inhibitor of IRAK-4. They have shown positive results in AML/MDS in spliceosome/FLT3 mutation patients as well as their non-target group. This will hopefully allow them to get accelerated approval with the FDA but they still have some hurdles to overcome. Longeveron is a cell therapy company looking to develop their Medicinal Signaling Cell therapy, Lomacel-B, in aging patients. They are focusing on Alzheimer's Disease, Aging Frailty as well...

Two CD47 targeting companies announced data updates from their clinical programs: Shattuk Labs and ALX Oncology. Both have suffered major stock price declines and in this episode, I go through the updates and discuss the potential of their molecules moving forward. Biogen cannot catch a break with the latest news continuing to contribute to new stock price lows. However, Medicare is close to announcing their National Coverage Determination (NCD) decision on Aduhelm, which will be a big mover...

Arvinas' PROTAC technology has led to two exciting molecules that are in the clinic for the treatment of breast cancer and prostate cancer. The company has spent years optimizing their protein degraders to achieve specific and effective protein degradation. ARV-471 targets the Estrogen Receptor for degradation and is in clinical trials for patients with HR+/HER2- breast cancer. The early efficacy and safety data bode very well for the company and an upcoming update will be provided in...

REGENXBIO ($RGNX) presents data using the Clearside Bio ($CLSD) SCS injector to treat wet AMD and diabetic retinopathy patients with RGX-314. Since existing treatments are on the market for these indications, it is very important that safety signals in these studies are reduced to a minimum. It is also critical that efficacy remains non-inferior to Eylea or Lucentis. In this video, I go through both readouts and discuss the potential for the therapy moving forward. 4D Molecular Therapeutics...

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 discovery and AI capabilities to the platform to create an end-to-end solution that can help Pharma streamline their preclinical and clinical programs....

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 payment as well as upcoming milestone payments. $RGNX/$CLSD are about to announce Cohort 1 data from their SCS wet AMD trial, which if positive,...

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 Sciences and the citizen's petition filing with the FDA. A package was provided to the FDA alleging misconduct and requesting a halt on...

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://www.patreon.com/breakingbiotech Follow me on twitter @matthewlepoire Send me an email [email protected] www.breakingbiotech.com...

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 $BCEL - 8:36 $AUPH - 15:57 $TGTX - 18:50 $KPTI - 24:00 $CRIS - 26:52 $BTAI - 30:20 $RGNX - 32:50 $MDGL - 36:39 $VKTX - 39:30 $SAVA - 41:18 $FDMT -...

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 companies: IONS, APLS, GMTX and CBIO. The major readout among them is that of APLS, happening in September and if successful, might bring new life into the...

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 readout, where we will get to see how effective Cosibelimab is in metastatic cutaneous squamous cell carcinoma. The company is also evaluating CK-101,...

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 eye diseases caused by neovascularization, discuss the sector as it stands today and explain my investment theses for each of those 3 companies. I...

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 efficacy, but have an impact on biomarkers. In this episode, I also touch on data updates provided by Curis and Replimune. Check out our NEW...

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 if this can be reproduced anywhere else. Oncternal is a targeted oncology company who submitted two abstracts to the 2021 ASCO conference. I go...

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 meeting in early June. They showed 8 out of 9 patients receiving some benefit from the treatment with 4 patients having a strong response (CR or CRi). In...