By David Bautz, PhD
Presentation of Case Study Involving AB-PA01
On April 3, 2018, AmpliPhi Biosciences Corp. (APHB) announced a case study involving a lung transplant patient that was administered AB-PA01 as part of a treatment for a resistant Pseudomonas aeruginosa lung infection was presented at the International Society for Hearth and Lung Transplantation 38th Annual Meeting. The case involved a 67-year old male patient that received a double lung transplant and then developed a recurring multi-drug resistant P. aeruginosa pneumonia. The patient was treated with AB-PA01 as an adjunctive treatment to antibiotics. Following treatment, the patient’s recurrent pneumonia stopped and commensal respiratory flora were re-established. This is an excellent example of the ability of bacteriophage therapy to treat recurrent infections while not having any long-term negative impact on the normal flora.
Positive Interim Results for Single-Patient Expanded Access Program
In January 2018, AmpliPhi announced positive interim results from the company’s single-patient expanded access program. A total of seven patients with serious or life-threatening infections, which were not responding to antibiotic therapy, were treated with AB-SA01 (n=4) or AB-PA01 (n=3). Six out of the seven patients (86%) achieved “treatment success”, which was defined as complete resolution or significant improvement of baseline signs and symptoms.
The company’s strategy is to use single patient expanded access protocols (Emergency IND in the U.S. and Special Access Scheme in Australia) to derive real-world data using AB-SA01 and AB-PA01 in patients suffering from severe, multidrug-resistant (MDR) infections. The following schematic shows the treatment schedule for the expanded access protocol. In order for a patient to qualify, they would need to have a serious or life-threatening infection that was not responding to antibiotics and that was also susceptible to either AB-SA01 or AB-PA01. Testing to confirm susceptibility to AB-SA01 or AB-PA01 can be performed in approximately 48 hours.
The characteristics of the seven patients reported on thus far are in the following slide. The APACHE II (Acute Physiology And Chronic Health Evaluation II) score is designed to measure the severity of disease of patients admitted to the intensive care unit (Knaus et al., 1985). The score (0-71) is calculated from the patient’s age and 12 physiological measurements, with a higher score signifying more severe disease and a higher risk of death.
Importantly, both AB-SA01 and AB-PA01 were safe and well-tolerated with no treatment-related serious adverse events (SAE). A total of 90 intravenous (IV) doses of AB-SA01 and a total of 402 IV and 92 inhaled doses of AB-PA01 were administered.
The following slide shows the clinical outcomes for the seven patients. Six of the seven were classified as “treatment success”, meaning that they had a complete resolution or significant improvement of baseline signs and symptoms while one patient failed treatment. The patient who failed presented with septic shock and an APACHE II score of 47 at baseline (which equates to a predicted mortality risk of 97%). The patient was not responding to antibiotic treatment and following three days of bacteriophage therapy they died during surgery on day 3. The treating physician deemed the death as unrelated to bacteriophage treatment.
The following slide shows that while there was one death during the first 28 days following treatment, there were no deaths reported in the Day 28-90 period, with some follow-ups still ongoing. We believe this is indicative of the bacteriophage treatment helping to eliminate the infection, and not just delay a negative outcome. The average APACHE II predicted mortality for the seven patients was 46% (with three patients predicted mortality >85%), thus having a mortality rate of only 14% in this group is very encouraging.
Additional Preclinical Studies for AB-SA01
On May 3, 2018, AmpliPhi announced it will use the Therapeutics Development Services funding by the National Institute of Allergy and Infectious Disease (NIAID) to conduct additional preclinical studies of AB-SA01. This program funds the necessary in vitro and in vivo preclinical studies that are required for advancing promising anti-infective therapeutics and applications are accepted by invitation only.
Collaborations for Expanded Access Programs
On March 13, 2018, AmpliPhi announced a Cooperative Research and Development Agreement (CRADA) with the U.S. Department of Veterans Affairs (VA) Palo Alto Health Care System in regards to expanded access to AB-SA01 and AB-PA01. The Principal Investigator for the collaboration is Mark Holodniy, MD, Professor of Medicine at Stanford University and the VA Palo Alto Health Care System. The CRADA will support the administration of AB-SA01 and AB-PA01 in patients at the VA Palo Alto Health Care System who have serious or life-threatening infections, do not respond to antibiotics, and meet the criteria for treatment under the FDA’s Expanded Access program.
On March 19, 2018, AmpliPhi announced a collaboration with the Western Sydney Local Health District and the Westmead Institute for Medical Research for the compassionate use of AB-SA01 and AB-PA01 in patients with severe infections. Several patients have been treated thus far at the Westmead Hospital under the single-patient expanded access program and this collaboration will allow for additional patients with serious or life-threatening infections to receive AB-SA01 and AB-PA01.
On May 15, 2018, AmpliPhi announced financial results for the first quarter of 2018. R&D expenses for the first quarter of 2018 and 2017 were $1.5 million, with an increase in clinical expenses in 2018 partially offset by a decrease in professional and consulting expenses. G&A expenses were $1.6 million for the first quarter of 2018 compared to $1.9 million for the first quarter of 2017. The decrease was primarily due to a decrease in legal and professional fees. Net loss for the first quarter of 2018 was $3.1 million, or $0.23 per share, compared to a net loss of $3.2 million, or $1.94 per share, in the first quarter of 2017.
As of March 31, 2018, the company had approximately $8.2 million in cash and cash equivalents. Cash burn during the first quarter of 2018 was $3.5 million. AmpliPhi has completed two public offerings thus far in 2018: In January 2018 the company sold 4.0 million shares of common stock at a price of $1.00 per share for gross proceeds of $4 million and in March 2018 the company sold 2.7 million shares of common stock at $1.10 per share for gross proceeds of $3.0 million. We anticipate the company has enough capital to fund operations into the fourth quarter of 2018. As of May 10, 2018, AmpliPhi had approximately 16.5 million shares of common stock outstanding and when factoring in in stock options and warrants a fully diluted share count of approximately 25.7 million.
We believe the data that AmpliPhi has compiled thus far from the expanded access program has been very encouraging. We continue to believe that the company will have compiled sufficient data by mid-2018 to approach the FDA about a registration pathway for AB-SA01 and AB-PA01 with a Phase 2 trial possibly starting in the fourth quarter of 2018. Our current valuation is $4.00 per share. However, we believe there is likely to be significant upside to our model once we learn more about what indications the company will be targeting later in 2018 for Phase 2 studies, at which time we may also get a better sense of timelines for approval.
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By David Bautz, PhD