RESEARCH REPORT UPDATE
BIOPHARMX (BPMX) REPORTS POSITIVE PHASE IIA DATA OF BPX-01 FOR ACNE
09/16/2016
By Grant Zeng, CFA
http://scr.zacks.com/News/Press-Releases/Pres...fault.aspx
NYSE:BPMX
BioPharmX (NYSE:BPMX) is developing BPX01, a non-lipophilic, topical antibiotic for the treatment of acne. BPX01 utilizes a transepidermal delivery mechanism for minocycline and other APIs that has the potential to kill p. acnes bacteria without the systemic side effects of orally-administered antibiotics.
The Phase IIa Completed
In March 2016, the company initiated a Phase IIa clinical study for BPX-01. The Phase IIa clinical study, conducted by KGL Skin Center, a Philadelphia-based independent clinical research facility, will assess the safety of BPX-01 in 30 patients randomized to 30 days of treatment with either BPX-01 or a placebo. The endpoints of this study include reduction of Propionibacterium acnes (P. acnes) and cutaneous tolerance of the BPX-01 formulation. This is the first clinical study in the clinical development program for BPX-01.
The principal investigator is Dr. Stuart Lessin, a board-certified dermatologist who serves as KGL's medical director.
On April 25, 2016, BioPharmX announced that it has completed enrollment for the Phase IIa study.
On August 22, 2016, the company released final findings from the Phase IIa study.
Daily application of BPX-01 resulted in a statistically significant reduction of P. acnes at four weeks compared to baseline. The reduction at four weeks was also statistically significant between BPX-01 and the vehicle control. No adverse cutaneous effects were observed, no clinically significant hematologic or chemistry alterations occurred, and no minocycline was detected in the plasma at any timepoint.
The reduction of P. acnes was 91 percent after four weeks of using BPX-01, a significant improvement for a topical formulation.
The reduction in P. acnes achieved in the Phase IIa study is similar to that reported in a 1996 study that effectively defined oral minocycline as the superior antimicrobial to fight P. acnes.
BPX-01 is the first topical gel formulation of minocycline that can penetrate the skin to deliver the antibiotic to the site of acne development in the pilosebaceous unit. BPX-01 is the first and only stable hydrophilic (non-oil-based) topical gel with fully solubilized minocycline. The study's patient exit survey indicated 100 percent satisfaction with BPX-01's usability and tolerability.
One recognized issue with oral minocycline is that – even though it is the antibiotic most commonly prescribed for the treatment of P. acnes – it enters the patient's bloodstream and can cause unwanted side effects. The BPX-01 Phase IIa study found no detectable levels of minocycline in the bloodstream of patients using the topical minocycline. The study also found no cutaneous toxicity and no adverse effects.
Further, results of a 28-day clinical PK study found no detectable minocycline in skin after two weeks of daily oral treatment with a 1-2 mg/kg dose, but did show minocycline concentrations in the plasma. A separate oral gavage versus BPX-01 minipig study also found minocycline in the plasma for minipigs in the oral group, but no minocycline in the plasma for the topical group. At the same time, the minipig study detected minocycline in the skin of the topical group, but found no minocycline in the skin of the oral group.
Phase IIb Initiated
Based on the positive Phase IIa data, on Aug. 24, 2016, BioPharmX announced that the first subject has been enrolled in its Phase IIb clinical trial assessing the efficacy and safety of BPX-01 for the treatment of acne vulgaris. The study is formally known as the OPAL (tOPicAL Minocycline Gel) study.
The 12-week, multi-center, double-blind, three-arm, vehicle controlled OPAL study expects to enroll 225 people, aged 9 to 40, who have moderate-to-severe inflammatory, non-nodular acne vulgaris. Safety will be assessed by physical examination, clinical laboratory tests, cutaneous tolerance scores and incidence of adverse events.
- The primary efficacy endpoint for the study is mean change from baseline in inflammatory lesion counts at Week 12.
- The secondary efficacy endpoint is achievement of at least a two-grade reduction in Investigator Global Assessment (IGA) at Week 12 compared to baseline.
The company expects results from the OPAL study in the first half of 2017.
The principal investigator is Joely Kaufman, a board certified dermatologist at The Skin Research Institute in Miami and fellow of the American Academy of Dermatology.
BPMX intends to pursue regulatory approval of BPX01 under Section 505(b)(2) of the FDC Act. The 505(b)(2) regulatory pathway may reduce the drug development risks and costs by using prior findings of safety and/or efficacy for an approved product. In BPX01 case, part of the safety and efficacy data from the oral formulation of minocycline may be used for the filing of a NDA for BPX01.
If everything goes well, we estimate BPX01 will be approved by the FDA in calendar 2019. Peak sales of BPX01 should be around $450 million.
BPX01’s Advantages over Oral Minocycline
All the side effects discussed above limit the use of oral minocycline for the treatment of acne. As a result, BioPharmX formulated BPX01 as a topical cream using a proprietary drug delivery technology. BPX01 is a new anhydrous, non-oil based topical antibiotic targeting acne bacteria.
BPX01 is designed to have several advantages compared to both orally-administered and other topically-administered retinoid- and antibiotic-based solutions. Advantages of BPX01 include:
- topical delivery of minocycline,
- controlled dosages targeted directly to affected area,
- increased delivery of API at low dosages,
- non-lipophilic design allowing for faster absorption by the skin,
- potential lower risk of systemic side effects common to orally administered antibiotics,
- Gel-like form rubs on like a sanitizer.
In addition, BPX01 has been shown in pre-clinical studies to possess anti-inflammatory properties, which reduce swelling and slow hyper-cornification.
During the third quarter, the company presented pre-clinical data at three key dermatology seminars suggesting that a 1% dose of minocycline in BPX-01 can work as well as the 4% topical products that are under development by competitors, lowering the likelihood of side effects.
Update on Fiscal Second Quarter Financials
For the second quarter ended July 31, 2016, total product revenue was $19,000, as compared to $5,000 for the same period of last year.
R&D expenses for the fiscal second quarter were $3.0 million, as compared to $1.1 million for the same quarter last year. The increase was primarily due to the increased headcount-related costs, preclinical, clinical studies and consulting expenses. In the second quarter of fiscal year 2017, the company completed the Phase IIa clinical study for BPX01, and in August 2016, began enrolling patients for the Phase IIb clinical study.
SG&A expenses for the fiscal second quarter were $2.1 million, as compared to $2.4 million for the same period last year. The decrease was primarily due to decreased advertising and promotional activities related to VI2OLET, offset by higher costs related to the market development activities of BPX01.
Total operating expenses for the current quarter were $5.0 million, compared with total operating expenses of $3.6 million in the prior fiscal year's second quarter. The increase in operating expenses resulted primarily from higher spending for the company's acne drug clinical trials.
Net loss for the second quarter was $5.0 million, or $0.18 per share, compared with a net loss of $3.7 million, or $0.24 per share, during the prior fiscal year's second quarter.
Excluding stock-based compensation expense and amortization of purchased intangible assets, non-GAAP net loss for the second quarter was $4.6 million, or $0.16 per share. During the second quarter of the prior fiscal year, the comparable non-GAAP net loss was $3.4 million, or $0.22 per share.
Cash and cash equivalents as of July 31, 2016 were $0.4 million.
After the end of the second quarter, the company raised approximately $3.1 million in capital through private offerings of its common stock and issuing convertible debt.
BIOPHARMX (BPMX) REPORTS POSITIVE PHASE IIA DATA OF BPX-01 FOR ACNE
09/16/2016
By Grant Zeng, CFA
http://scr.zacks.com/News/Press-Releases/Pres...fault.aspx
NYSE:BPMX
BioPharmX (NYSE:BPMX) is developing BPX01, a non-lipophilic, topical antibiotic for the treatment of acne. BPX01 utilizes a transepidermal delivery mechanism for minocycline and other APIs that has the potential to kill p. acnes bacteria without the systemic side effects of orally-administered antibiotics.
The Phase IIa Completed
In March 2016, the company initiated a Phase IIa clinical study for BPX-01. The Phase IIa clinical study, conducted by KGL Skin Center, a Philadelphia-based independent clinical research facility, will assess the safety of BPX-01 in 30 patients randomized to 30 days of treatment with either BPX-01 or a placebo. The endpoints of this study include reduction of Propionibacterium acnes (P. acnes) and cutaneous tolerance of the BPX-01 formulation. This is the first clinical study in the clinical development program for BPX-01.
The principal investigator is Dr. Stuart Lessin, a board-certified dermatologist who serves as KGL's medical director.
On April 25, 2016, BioPharmX announced that it has completed enrollment for the Phase IIa study.
On August 22, 2016, the company released final findings from the Phase IIa study.
Daily application of BPX-01 resulted in a statistically significant reduction of P. acnes at four weeks compared to baseline. The reduction at four weeks was also statistically significant between BPX-01 and the vehicle control. No adverse cutaneous effects were observed, no clinically significant hematologic or chemistry alterations occurred, and no minocycline was detected in the plasma at any timepoint.
The reduction of P. acnes was 91 percent after four weeks of using BPX-01, a significant improvement for a topical formulation.
The reduction in P. acnes achieved in the Phase IIa study is similar to that reported in a 1996 study that effectively defined oral minocycline as the superior antimicrobial to fight P. acnes.
BPX-01 is the first topical gel formulation of minocycline that can penetrate the skin to deliver the antibiotic to the site of acne development in the pilosebaceous unit. BPX-01 is the first and only stable hydrophilic (non-oil-based) topical gel with fully solubilized minocycline. The study's patient exit survey indicated 100 percent satisfaction with BPX-01's usability and tolerability.
One recognized issue with oral minocycline is that – even though it is the antibiotic most commonly prescribed for the treatment of P. acnes – it enters the patient's bloodstream and can cause unwanted side effects. The BPX-01 Phase IIa study found no detectable levels of minocycline in the bloodstream of patients using the topical minocycline. The study also found no cutaneous toxicity and no adverse effects.
Further, results of a 28-day clinical PK study found no detectable minocycline in skin after two weeks of daily oral treatment with a 1-2 mg/kg dose, but did show minocycline concentrations in the plasma. A separate oral gavage versus BPX-01 minipig study also found minocycline in the plasma for minipigs in the oral group, but no minocycline in the plasma for the topical group. At the same time, the minipig study detected minocycline in the skin of the topical group, but found no minocycline in the skin of the oral group.
Phase IIb Initiated
Based on the positive Phase IIa data, on Aug. 24, 2016, BioPharmX announced that the first subject has been enrolled in its Phase IIb clinical trial assessing the efficacy and safety of BPX-01 for the treatment of acne vulgaris. The study is formally known as the OPAL (tOPicAL Minocycline Gel) study.
The 12-week, multi-center, double-blind, three-arm, vehicle controlled OPAL study expects to enroll 225 people, aged 9 to 40, who have moderate-to-severe inflammatory, non-nodular acne vulgaris. Safety will be assessed by physical examination, clinical laboratory tests, cutaneous tolerance scores and incidence of adverse events.
- The primary efficacy endpoint for the study is mean change from baseline in inflammatory lesion counts at Week 12.
- The secondary efficacy endpoint is achievement of at least a two-grade reduction in Investigator Global Assessment (IGA) at Week 12 compared to baseline.
The company expects results from the OPAL study in the first half of 2017.
The principal investigator is Joely Kaufman, a board certified dermatologist at The Skin Research Institute in Miami and fellow of the American Academy of Dermatology.
BPMX intends to pursue regulatory approval of BPX01 under Section 505(b)(2) of the FDC Act. The 505(b)(2) regulatory pathway may reduce the drug development risks and costs by using prior findings of safety and/or efficacy for an approved product. In BPX01 case, part of the safety and efficacy data from the oral formulation of minocycline may be used for the filing of a NDA for BPX01.
If everything goes well, we estimate BPX01 will be approved by the FDA in calendar 2019. Peak sales of BPX01 should be around $450 million.
BPX01’s Advantages over Oral Minocycline
All the side effects discussed above limit the use of oral minocycline for the treatment of acne. As a result, BioPharmX formulated BPX01 as a topical cream using a proprietary drug delivery technology. BPX01 is a new anhydrous, non-oil based topical antibiotic targeting acne bacteria.
BPX01 is designed to have several advantages compared to both orally-administered and other topically-administered retinoid- and antibiotic-based solutions. Advantages of BPX01 include:
- topical delivery of minocycline,
- controlled dosages targeted directly to affected area,
- increased delivery of API at low dosages,
- non-lipophilic design allowing for faster absorption by the skin,
- potential lower risk of systemic side effects common to orally administered antibiotics,
- Gel-like form rubs on like a sanitizer.
In addition, BPX01 has been shown in pre-clinical studies to possess anti-inflammatory properties, which reduce swelling and slow hyper-cornification.
During the third quarter, the company presented pre-clinical data at three key dermatology seminars suggesting that a 1% dose of minocycline in BPX-01 can work as well as the 4% topical products that are under development by competitors, lowering the likelihood of side effects.
Update on Fiscal Second Quarter Financials
For the second quarter ended July 31, 2016, total product revenue was $19,000, as compared to $5,000 for the same period of last year.
R&D expenses for the fiscal second quarter were $3.0 million, as compared to $1.1 million for the same quarter last year. The increase was primarily due to the increased headcount-related costs, preclinical, clinical studies and consulting expenses. In the second quarter of fiscal year 2017, the company completed the Phase IIa clinical study for BPX01, and in August 2016, began enrolling patients for the Phase IIb clinical study.
SG&A expenses for the fiscal second quarter were $2.1 million, as compared to $2.4 million for the same period last year. The decrease was primarily due to decreased advertising and promotional activities related to VI2OLET, offset by higher costs related to the market development activities of BPX01.
Total operating expenses for the current quarter were $5.0 million, compared with total operating expenses of $3.6 million in the prior fiscal year's second quarter. The increase in operating expenses resulted primarily from higher spending for the company's acne drug clinical trials.
Net loss for the second quarter was $5.0 million, or $0.18 per share, compared with a net loss of $3.7 million, or $0.24 per share, during the prior fiscal year's second quarter.
Excluding stock-based compensation expense and amortization of purchased intangible assets, non-GAAP net loss for the second quarter was $4.6 million, or $0.16 per share. During the second quarter of the prior fiscal year, the comparable non-GAAP net loss was $3.4 million, or $0.22 per share.
Cash and cash equivalents as of July 31, 2016 were $0.4 million.
After the end of the second quarter, the company raised approximately $3.1 million in capital through private offerings of its common stock and issuing convertible debt.
