1/ $CABA Cabaletta Bio
A biotech company that can potentially generate cures for patients with B cell autoimmune diseases.
A biotech company that can potentially generate cures for patients with B cell autoimmune diseases.
2/ The company is focused on CAAR-T for B cell mediated autoimmune diseases. They are the only player in this space currently as many of the CAR-T companies are focused on oncology.
3/ Traditional CAR-T therapies kill all of a patients B cells expressing CD19 because they can’t differentiate good from bad. Cabalatta’s treatment will only kill about 1% of the patients B cells as these are the only ones that cause disease.
4/ The lead indication is in Pemphigus Vulgaris (PV), a rare diseases that causes blisters in the mouth. Treatment requires Rituximab plus steroids (~3,500 mg/yr). Often this leads to temporary relief but then the patients relapse several months later.
5/ Below is the trial design for PV. Enrollment has been fast because the FDA allowed them to only wait 1 week in between patients instead of 2 as the original protocol specified. They are currently enrolling patients in the 500M dose cohort.
6/ Evaluation criteria for trial readouts
• Persistence of DSG3-CAART detected via qPCR
• Change in level of DSG3 antibodies (targeting persistent reduction)
• Reduced mPV therapy and absence of new systemic rescue therapy
• Clinical symptoms of the patient
• Persistence of DSG3-CAART detected via qPCR
• Change in level of DSG3 antibodies (targeting persistent reduction)
• Reduced mPV therapy and absence of new systemic rescue therapy
• Clinical symptoms of the patient
7/ Strong operating partnership with Penn CVPF manufacturing organization. Use of validated process from CAR T experience at Penn helps mitigate risk. They have had a 100% success rate for DesCAARTesTM trial manufacturing to date.
8/ Potential to report biologic activity (20M & 100M) and 28-day safety data (500M) in 4Q21. IF IF IF this shows efficacy expect a large move in the SP because there is read through to most of the other indications in the pipeline.
9/ It is possible that these doses are still too low. They started very low for safety reasons even though this treatment should have a very safe profile. They may need to go all the way up to 2.5B cells or even higher. They can also potentially give repeat dosing if necessary.
10/ Financials
Current market cap 288M
25M shares outstanding
Cash runway through 4Q 22 with 133M in cash and investments as of June 2021
Current market cap 288M
25M shares outstanding
Cash runway through 4Q 22 with 133M in cash and investments as of June 2021
11/ Overall there is still a lot of unknowns with this company but there are several near term catalysts that could send the SP much higher. Based on what I have read and heard from management so far this is a potential #gamechanger and I currently own shares.
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