| Basic Facts for Quarter Ending June 30, 2019 (In Thousands unless otherwise noted) | |||
| Q2-2019 | Q1-2019 | Q2-2018 | |
| Grants & Other Revenue | 410 | 231 | 287 |
| Total Income | 410 | 231 | 404 |
| R&D Expense | 1,644 | 1,811 | 3,389 |
| G&A Expenses | 832 | 976 | 1,178 |
| Total Operating Expenses | 2,476 | 2,788 | 4,567 |
| Operating Income (Loss) | (2,066) | (2,557) | (4,163) |
| Net Income (Loss) | (2,519) | (2,519) | (4,124) |
| Comprehensive Income (Loss) Inc. gains/losses on securities | (2,046) | (2,532) | (4,126) |
| Basic & diluted profit (loss) per share | (0.59) (0.59) | (0.08) (0.08) | (1.42) (1.42) |
| Avg. Shares Outstanding (Basic) | 3,458 | 3,290 | 2,903 |
| Recent Price (per share) | $3.75 (8/6/19) | $4.20 (5/13/19) | $12.10 (7/27/18) |
| Market Capitalization | 13M | 14M | 35M |
| Cash & Short-term Investments | $5,891 | $7,170 | $6,339 |
| Marketable Securities | - | 185 | 5,995 |
| CAP-1002 –Duchenne Muscular Dystrophy & Adult Heart Diseases Cardiac tissue is extracted from cadavers and then explants are extracted and cultured in the lab. The aggregated cells are cardiospheres. These cardiospheres are efficiently multiplied and implanted into the patients. These implanted cells are called “cardiosphere-derived cells” or CDCs. | |
| Duchenne Muscular Dystrophy – HOPE-2 Trial - Repeat Dosing & Chronic Administration - Skeletal Muscle Function Capricor is expanding from the heart to skeletal muscle recovery in DMD patients. This will require systemic infusion, and thus a greater dose. The recent interim study results showed that a single dose to the heart results provides measurable advantages at 3 months, and that it largely dissipates at 6 months.The trial redoses every 3 months. The primary endpoint is based on the middle PUL (Performanceof the UpperLimb) scores at 12 months. The animal models did not display a meaningful immune response to the allogeneic cells. It will be crucial for the “booster shots” to show safety and prolonged efficacy. The ongoing trial, HOPE-2, has a randomized, double-blind, placebo-controlled design The majority of the 18 DMD patients are early-stage non-ambulatory (wheelchair). The redosing interval will be 3-months.The mid-PUL test at baseline vs. 12 months is the primary efficacy endpoint. Capricor received an RMAT designation as well as Orphan and Rare Pediatric Disease designations from the FDA. | |
| Q4-2019 | |
| Receive 150M cells of CAP-1002 or Placebo at every 3 months | 1H-2020 |
| Independent Investigator Trials – Adult Heart Diseases Capricor’s co-Founder, Eduardo Marban, is the lead investigator in these trials. He is the Cardiology Chief at Cedars-Sinai Medical Center in West Los Angeles. The studies advance the clinical development of CAP-1002 in different cardiovascular diseases: heart failure with preserved ejection fraction and pulmonary arterial hypertension (PAH). In both trials, CAP-1002 is infused through the coronary arteries. With good results, Capricor may advance these indications through in-house programs. | |
| Top-line Results – Phase 2a Regress-HFPEF Trial – 40 Pts with Heart Failure with Preserved Ejection Fraction – Randomized, double-blind, placebo-controlled study. The endpoints focus on functional measures as well as MRI-assessed fibrosis. | January 2020* |
| Top-line Results – Phase 1a / 1b ALPHA trial – 26 PAH patients The open-label, single-arm dose escalation phase 1a part has 6 patients Double-blind, placebo-controlled Phase 1b part has 20 patients | 1H-2020 |
| CAP-2003 (Exosomes) – Organ Diseases Exosomes serve as a primitive, robust way for intercellular communication.It is a lipid bilayer that is secreted by cells and in Capricor’s case, it contains some microRNA growth factors that transmit regenerative instructions to cells.The goal is to recreate the rejuvenation and tissue repair of present stem-cell therapies without its associated hazards (e.g. immunogenicity). Capricor and affiliated scientists have established that the exosomes are the regenerative source within CAP-2003. Exosomes elicit a weaker immune response than CAP-2003 and have a lower cost of goods coupled with a greater ease of manufacturing. CAP-2003 is an especially promising (future) candidate for retreatment dosing in future trials. | |
| Hypoplastic Left Heart Syndrome (HLHS) HLHS occurs in about 1,000 patients in the United States. It is a serious, pediatric orphan disease. In Phase I and II trials, Japanese investigators reportedsuccessusingcardiosphere-derived cells(CDCs) in HLHS patients. This provides proof of concept as Capricor advances its CDC-derived exosomes. | |
| IND Submission | TBA |
| Launch Phase 1 Trial | TBA |
| Inflammatory Disease TBA | |
| IND Submission | TBA |
| U.S. Army Institute of Surgical Research (USAISR) The military surgical research team is studying CAP-2003 for possible use with severely injured soldiers. It desires a formulation that is stable at room temperature, which implies a lyophilized (freeze-dried) preparation. Capricor may then receive solid data that extends well beyond the usual reach for a Phase 1 trial. | |
| Ongoing Research & Clinical Application | TBA |
Original Article: Capricor Therapeutics - Q2-2019 Update