Investors love biotech stocks for the lottery ticket-like returns they can offer if a company strikes medical gold. Case in point: Ampliphi Biosciences Corp (NYSEAMERICAN:APHB), whose shares are jumping nearly 16% on Thursday.
The biotechnology firm announced positive topline results for the first seven patients treated under its ongoing single-patient expanded access program. Six of the seven patients (86%) achieved treatment success (physician’s assessment), defined as complete resolution or significant improvement of baseline signs and symptoms. All patients were severely ill and unresponsive to antibiotic treatment at the time of enrollment.
AmpliPhi CEO Paul C. Grint: “I am very encouraged by these initial results for treatment with AB-SA01 or AB-PA01 in severely ill patients who were not responding to antibiotics […] We look forward to treating up to an additional 20 patients during the first half of 2018 and discussing our findings with the FDA to determine a development path forward to registration. Our ongoing dialogue with infectious disease thought leaders continues to indicate growing support for our program.”
Dr. Jonathan Iredell, Professor of Medicine and Microbiology at the University of Sydney: “Bacteriophage therapeutics have the potential to be a safe and potent modality for treating serious bacterial infections, and also provide an option for those with antibiotic resistant or relapsing infection. Encouraging initial results make it important to proceed to full clinical evaluation and further development of this therapeutic approach.”
Robert T. Schooley, Professor in the infectious disease division at the University of California, San Diego: “The treatment with bacteriophages of severely ill patients with antibiotic-resistant infections warrants further clinical investigation and holds promise as a new approach to this critical unmet medical need […] The FDA also recognizes that multi-drug resistant infections are a real problem and I believe they see this approach as one that clearly needs to be evaluated.”
AmpliPhi COO Igor P. Bilinsky: “Bacteriophage therapies have been around for the past hundred years […] It is only now, enabled by advances in biologics manufacturing and DNA sequencing, that we are able to produce GMP grade phage products that could be suitable for intravenous administration. This is an important step for developing phages as a novel, precisely targeted therapeutic modality for patients with serious infections who have few or no other treatment options and for helping humanity solve the growing crisis of antibiotic resistance.”
Expanded Access Program Design and Topline Results
The expanded access approach allows critically ill patients to receive experimental, unapproved therapies in an attempt to save lives. Severely ill patients can receive treatment in the U.S. under an emergency IND and in Australia under the Special Access Scheme. AmpliPhi’s lead product candidates, AB-SA01, for Staphylococcus aureusinfections, and AB-PA01, for Pseudomonas aeruginosa infections, are being provided through this program. Among the first seven patients treated, four patients received intravenous AB-SA01 and three received AB-PA01 administered intravenously and in some cases as an inhaled therapy. Bacteriophage treatment was administered along with the treating physician’s choice of best available antibiotic therapy. Treated patients suffered from bacteremia, endocarditis and lung infections, and both investigational products were well tolerated in all patients with no treatment-related serious adverse events reported.
Treatment success, defined as complete resolution or significant improvement of baseline signs and symptoms, was reported in six out of seven patients (86%) by physician’s assessment. One patient was determined to be a treatment failure due to death, which occurred during surgery after three days of bacteriophage treatment. The treating physician determined that the one death was unrelated to treatment with bacteriophage therapy. The 28-day all-cause mortality rate was 14%. No additional deaths occurred up to 90 days following initiation of therapy, and patient follow up is continuing. Based on the APACHE II scores (a validated critical care scoring system predictive of mortality) of the seven patients prior to initiation of bacteriophage therapy, the predicted mortality rate for this patient group was 46%.
No bacterial isolates resistant to the bacteriophage therapeutics were detected during the bacteriophage treatment course. Additional analyses of these data are ongoing, and presentations or publications of the detailed results are planned.
On the ratings front, H.C. Wainwright analyst Joseph Pantginis reiterated a Buy rating on APHB, with a price target of $5.00, in a report issued on November 15. The current price target implies an upside of 294% from current levels. According to TipRanks.com, Pantginis has a yearly average loss of 18.3%, a 33% success rate, and is ranked #4710 out of 4750 analysts.
AmpliPhi operates as a biopharmaceutical company, which engages in the discovery, development, and commercialization of novel phage therapeutics. It operates in segments of the biotechnology and biopharmaceutical markets.