PRISM Q&A CORNER
Jonathan Kaufman, Lipella PharmaceuticalsLipella Pharmaceuticals: Expanded Clinical Pipeline Targets Significant Unmet Need
Clinical stage pharmaceutical company, Lipella Pharmaceuticals (Nasdaq: LIPO) was recently granted Orphan Drug Designation by the FDA for a second drug candidate targeting the serious rare disease, oral Graft-versus-Host Disease (GvHD).
In 2023, the company received Orphan Drug Designation for LP-10, a potential treatment for hemorrhagic cystitis. With these nods from the FDA, the company is on track to deliver two safe and effective treatments to cancer survivors, with a third therapeutic in its pipeline. The company’s clinical pipeline has strengthened and expanded significantly over the past 12 months, and offers a strong value proposition for investors.
We spoke to Jonathan Kaufman, Lipella’s Chief Executive Officer, about the company’s recent regulatory accomplishments and how investors stand to benefit.
A:We are currently developing candidates for three indications. The first is hemorrhagic cystitis, which is serious, potentially life threatening bladder damage that can occur months or even years after radiation treatment for pelvic cancers, or bladder-toxic chemotherapy. We are also developing a treatment for oral lichen planus, a disease characterized by pain, redness and swelling inside the mouth, that affects over six million Americans. Our third indication is oral Graft-versus-Host-Disease (GvHD), a serious rare disease that causes significant pain and discomfort, making it difficult for patients to eat, drink and speak.
Q:A:While patient populations are low, orphan drugs have higher pricing power and can be highly profitable in spite of these smaller addressable market sizes. The global orphan drug market is projected to grow from $170.49 billion in 2023 to $368.43 billion by 2030, at a CAGR of 11.6%. In the case of hemorrhagic cystitis, Lipella’s expected annual revenue per patient is $20,000 and our peak annual revenue estimates exceed $1 billion. The disease has an addressable market of ~60,000 potential new patients each year. Oral lichen planus has a patient population of six million in the United States alone, and GvHD affects up to half of all patients who receive an allogeneic stem cell transplant, with up to 83% of GvHD patients developing oral GvHD.
Q:A: Tacrolimus is a safe and effective drug with well understood pharmacologic mechanisms. It is a potent vasoconstrictor and has strong anti-inflammatory properties, making it an excellent candidate for Lipella’s three disease areas. Our strategy involves leveraging the 505(b)(2) pathway which we believe will lower cost and allow for a faster approval process by allowing us to bypass Phase 1 trials and immediately initiate Phase 2 clinical trials. It also potentially mitigates risk from a CMC, safety and clinical development standpoint, and offers greater flexibility from the FDA.
Q:A:Orphan drug development can often be more profitable, entail lower risk and result in faster time to approval than non-orphan therapeutics. We are excited to have been granted Orphan Drug Designation for LP-10 in the treatment of hemorrhagic cystitis, and for LP-410 for oral GvHD. These serious rare diseases have a significant impact on patients’ lives and currently have no approved treatment. Our goal is to deliver safe and effective treatments as quickly as possible to the patients who need them most.
Clinical stage pharmaceutical company, Lipella Pharmaceuticals (Nasdaq: LIPO) was recently granted Orphan Drug Designation by the FDA for a second drug candidate targeting the serious rare disease, oral Graft-versus-Host Disease (GvHD).
In 2023, the company received Orphan Drug Designation for LP-10, a potential treatment for hemorrhagic cystitis. With these nods from the FDA, the company is on track to deliver two safe and effective treatments to cancer survivors, with a third therapeutic in its pipeline. The company’s clinical pipeline has strengthened and expanded significantly over the past 12 months, and offers a strong value proposition for investors.
We spoke to Jonathan Kaufman, Lipella’s Chief Executive Officer, about the company’s recent regulatory accomplishments and how investors stand to benefit.
We are currently developing candidates for three indications. The first is hemorrhagic cystitis, which is serious, potentially life threatening bladder damage that can occur months or even years after radiation treatment for pelvic cancers, or bladder-toxic chemotherapy. We are also developing a treatment for oral lichen planus, a disease characterized by pain, redness and swelling inside the mouth, that affects over six million Americans. Our third indication is oral Graft-versus-Host-Disease (GvHD), a serious rare disease that causes significant pain and discomfort, making it difficult for patients to eat, drink and speak.
While patient populations are low, orphan drugs have higher pricing power and can be highly profitable in spite of these smaller addressable market sizes. The global orphan drug market is projected to grow from $170.49 billion in 2023 to $368.43 billion by 2030, at a CAGR of 11.6%. In the case of hemorrhagic cystitis, Lipella’s expected annual revenue per patient is $20,000 and our peak annual revenue estimates exceed $1 billion. The disease has an addressable market of ~60,000 potential new patients each year. Oral lichen planus has a patient population of six million in the United States alone, and GvHD affects up to half of all patients who receive an allogeneic stem cell transplant, with up to 83% of GvHD patients developing oral GvHD.
Tacrolimus is a safe and effective drug with well understood pharmacologic mechanisms. It is a potent vasoconstrictor and has strong anti-inflammatory properties, making it an excellent candidate for Lipella’s three disease areas. Our strategy involves leveraging the 505(b)(2) pathway which we believe will lower cost and allow for a faster approval process by allowing us to bypass Phase 1 trials and immediately initiate Phase 2 clinical trials. It also potentially mitigates risk from a CMC, safety and clinical development standpoint, and offers greater flexibility from the FDA.
Orphan drug development can often be more profitable, entail lower risk and result in faster time to approval than non-orphan therapeutics. We are excited to have been granted Orphan Drug Designation for LP-10 in the treatment of hemorrhagic cystitis, and for LP-410 for oral GvHD. These serious rare diseases have a significant impact on patients’ lives and currently have no approved treatment. Our goal is to deliver safe and effective treatments as quickly as possible to the patients who need them most.
