Pathalys Pharma and Launch Therapeutics Announce First Patient Enrolled Ahead of Schedule in Pivotal Phase 3 Program for Upacicalcet in Patients Receiving Hemodialysis

Fort Developer on May 31, 2023

Pathalys and LaunchTx leverage recently announced collaboration to accelerate enrollment for the upacicalcet development program.

Research Triangle Park, North Carolina and Boston, Massachusetts, May 31, 2023 – Pathalys Pharma, Inc., a private, late-stage biopharma company co-founded by Catalys Pacific and DaVita Venture Group, and Launch Therapeutics (Launch Tx), a clinical development company, today announced that the first patient has been enrolled in the PATH (PTH Attenuation Trial in Hemodialysis) clinical program, consisting of two identical phase 3 trials, to assess upacicalcet for the treatment of secondary hyperparathyroidism (SHPT) in end-stage kidney disease (ESKD) patients on hemodialysis

The PATH clinical trial program is the output of Pathalys’ previously disclosed intent to initiate two identical phase 3 clinical trials to evaluate upacicalcet. Upacicalcet is a novel calcimimetic that performed exceptionally well in phase 3 studies in Japan where it was approved in 2021 for the treatment of SHPT in patients on dialysis.

As part of its strategic collaboration with Pathalys, Launch Tx has the lead role for operational execution and management of the clinical trials and Pathalys has primary responsibility for PATH study design, manufacturing, FDA regulatory interactions, medical oversight, and commercialization.

“We have enrolled our first patient for this program ahead of schedule. With the successful conclusion of this trial, we hope to accelerate upacicalcet toward registration in the U.S. with the goal of making this therapy available for dialysis patients with SHPT.” said Dr. Theodore Danoff, Chief Medical Officer and SVP, Clinical Development at Pathalys. “The advancement of an intravenous calcimimetic like upacicalcet may allow patients on dialysis better control of parathyroid hormone levels in SHPT in a convenient and compliant way while also lowering risk of upper gastrointestinal adverse events and hypocalcemia, which continue to be an issue with current calcimimetics.”

Jessica Kendrick, MD, MPH, the principal investigator (PI) for the studies, commented, “Advancing upacicalcet will be a very meaningful step for patients and enrolling the first patient ahead of schedule is promising given that the sooner we can dose our first patient, the closer we will be to obtaining the needed data for FDA review and hopefully approval.”

“The combination of Pathalys Pharma and Launch Therapeutics is already demonstrating immense value with respect to the speed and efficiency of this program. I firmly believe that the operational expertise and immediate resources provided by the Launch Tx team has contributed to meeting this key milestone ahead of schedule.” said Elisha Talley-Roithner, Head of Operations at Launch Tx.

The PATH study program is designed to assess the efficacy of upacicalcet as measured by its ability to reduce intact parathyroid hormone (iPTH) by 30 percent or more in participants with secondary hyperparathyroidism (SHPT) and currently on hemodialysis.

Each trial will randomize approximately 375 patients (2:1) to upacicalcet or placebo. Eligible patients will be adults receiving in-center hemodialysis three times weekly and whose iPTH values are 450 pg/mL or greater. Upacicalcet will be titrated based on iPTH and serum corrected calcium (cCa) levels with dose steps between 25 and 300 mcg as a bolus IV injection given at the end of a dialysis session. Doses will be titrated to maintain iPTH in the range of 150-300 pg/mL.

 

About Pathalys Pharma

Pathalys Pharma, Inc. is a private, late-stage clinical biopharmaceutical company committed to the development of multiple advanced therapeutics that address unmet needs in the management of ESKD. Pathalys’ initial asset is upacicalcet, a novel calcimimetic with the potential to improve the treatment of SHPT in hemodialysis patients. Beyond upacicalcet, Pathalys continues to identify other high priority needs and potential solutions for patients with ESKD. Pathalys was co-founded by Catalys Pacific and DaVita Venture Group and is headquartered in Research Triangle Park, North Carolina.

 

For more information about Pathalys, please visit pathalys.com.

 

About Launch Therapeutics

Launch Therapeutics (Launch Tx) is a clinical development company with a mission to disrupt the late-stage development paradigm, accelerate timelines to regulatory success, and bring new medicines to patients faster. Launch Tx offers pharmaceutical and biotech companies a variety of innovative models that combine access to capital with deep drug development, medical, clinical operations, regulatory, and commercialization expertise. These models include significant risk financing, clinical co-development, and full in-licensing, all of which offer partners an aligned and efficient approach to realizing the potential of late-stage clinical programs across any therapeutic area. Founded in 2022, Launch Tx is backed by leading investors, Carlyle and its life sciences franchise, Abingworth, and is led by a committed, experienced team with an enthusiastic passion to fulfill its mission.

 

For more information, visit launchtx.com and follow us on LinkedIn.

 

Contact Information

For Pathalys
Media and Investor Contact:
Patrick Treanor
ptreanor@pathalys.com

 

For Launch Tx
Media Contact:
Elizabeth Kuronen
elizabeth.kuronen@launchtx.com

 

 

Pathalys Pharma Raises $150 Million in Secured Product Financing and Equity led by Abingworth; Forms Strategic Collaboration with Launch Therapeutics to Advance Upacicalcet through Pivotal Phase 3 Studies

Fort Developer on January 18, 2023

  • Abingworth, a leading transatlantic life sciences investment firm and part of global investment firm Carlyle (NASDAQ: CG), led financings and was joined by Carlyle, OrbiMed, and Pathalys’ founding investors Catalys Pacific and DaVita Venture Group
  • Transaction is expected to fully fund upacicalcet through pivotal phase 3 studies and pre-commercialization activities
  • Collaboration leverages clinical development experience of Launch Tx and Pathalys’ extensive dialysis expertise to advance the upacicalcet development program

Research Triangle Park, North Carolina and Boston, Massachusetts, January 18, 2023  — Pathalys Pharma, Inc., a private, late-stage biopharma company co-founded by Catalys Pacific and DaVita Venture Group, and Launch Therapeutics (Launch Tx), a clinical development company, today announced a collaboration to advance phase 3 clinical trials for upacicalcet (designated PLS240 for clinical trials) for the treatment of secondary hyperparathyroidism (SHPT) in patients on hemodialysis. Launch Tx is backed by funds managed by global investment firm Carlyle (NASDAQ: CG) and its life sciences franchise Abingworth.

Pathalys also announced it has raised $150 million through a combination of secured product financing and equity to support the two phase 3 clinical trials, registration efforts and pre-commercialization activities for upacicalcet. Abingworth led both financings and was joined by Carlyle and OrbiMed, along with Pathalys’ founding investors Catalys Pacific and DaVita Venture Group. Pathalys retains worldwide commercial rights to upacicalcet, outside Japan and Asia, and has the option to prepay its obligations in full at any time.

“Pathalys is very excited to collaborate with Launch Tx and to have the backing of such an esteemed group of healthcare investors. Through this collaboration and transaction, Pathalys expects to be fully funded through the completion of its planned phase 3 trials, and pre-commercialization efforts for upacicalcet.” said Neal Fowler, Chief Executive Officer at Pathalys. “We look forward to working closely with Launch Tx to advance upacicalcet toward registration in the U.S. with the goal of making this important product candidate available for dialysis patients with SHPT.”

The co-development agreement creates a collaboration that combines the exceptional clinical development resources available through Launch Tx and the extensive dialysis expertise of Pathalys to advance the upacicalcet clinical development program. Launch Tx will have the lead role in the operational execution and management of the planned clinical trials and Pathalys will retain primary responsibility for manufacturing, FDA regulatory interactions and commercialization.

Anshul Thakral, CEO of Launch Tx, commented, “We are thrilled to collaborate with Pathalys to advance upacicalcet. Launch Tx will bring deep clinical and operational resources to this phase 3 program to accelerate the path to regulatory approval. We offer a unique approach that helps both pharma and biotech companies develop their late-stage therapies and plan to demonstrate that approach through our collaboration with Pathalys.”

Earlier this year, Pathalys disclosed its plans to initiate two phase 3 clinical trials to evaluate upacicalcet as a treatment alternative to currently available calcimimetics. Upacicalcet is a novel calcimimetic that performed exceptionally well in phase 3 studies in Japan where it was approved last year for the treatment of SHPT in patients on dialysis. An intravenous calcimimetic like upacicalcet may allow patients on dialysis better control of serum parathyroid hormone levels in SHPT in a convenient and compliant way, without increasing their calcium-phosphorus production while, more importantly, lowering their risk for vascular calcifications.

About Upacicalcet

Upacicalcet is an intravenously administered investigational small molecule calcimimetic that may control intact parathyroid hormone (iPTH) levels in patients with secondary hyperparathyroidism (SHPT) receiving hemodialysis. Upacicalcet was granted Marketing Authorization in Japan in 2021, under the brand name UPASITA®, based on a phase 3, 24-week double-blind, placebo-controlled dose-titration study and a second phase 3, 52-week open-label, dose-titration study. In these studies, upacicalcet was shown to significantly reduce iPTH and maintain long-term iPTH suppression, as well as demonstrate the potential for fewer adverse events (AEs) than currently available therapies.

About Pathalys Pharma

Pathalys Pharma, Inc. is a private, late-stage clinical biopharmaceutical company committed to the development of multiple advanced therapeutics that address unmet needs in the management of end-stage kidney disease (ESKD). Pathalys’ initial asset is upacicalcet, a novel calcimimetic with the potential to improve the treatment of SHPT in hemodialysis patients. Beyond upacicalcet, Pathalys continues to identify other high priority needs and potential solutions for patients with ESKD. Pathalys was co-founded by Catalys Pacific and DaVita Venture Group and is headquartered in Research Triangle Park, North Carolina.

For more information about Pathalys, please visit pathalys.com.

About Launch Therapeutics

Launch Therapeutics (Launch Tx) is a clinical development company with a mission to disrupt the late-stage development paradigm, accelerate timelines to regulatory success, and bring new medicines to patients faster. Launch Tx offers pharmaceutical and biotech companies a variety of innovative models that combine access to capital with deep drug development, medical, clinical operations, regulatory, and commercialization expertise. These models include significant risk financing, clinical co-development, and full in-licensing, all of which offer partners an aligned and efficient approach to realizing the potential of late-stage clinical programs across any therapeutic area. Founded in 2022, Launch Tx is backed by leading investors, Carlyle and its life sciences franchise, Abingworth, and is led by a committed, experienced team with an enthusiastic passion to fulfill its mission.

For more information, visit launchtx.com and follow us on LinkedIn.

Contact Information

For Pathalys
Media and Investor Contact:
Patrick Treanor
ptreanor@pathalys.com

For Launch Tx
Media Contact:
Elizabeth Kuronen
elizabeth.kuronen@launchtx.com

Pathalys Pharma to Present at the 21st Annual Needham Virtual Healthcare Conference

Fort Developer on April 9, 2022

Research Triangle Park, N.C., April 8, 2022 – Pathalys Pharma, Inc., a private, late-stage biopharma company committed to the development and commercialization of a range of best-in-class therapies for patients with end stage kidney disease (ESKD), today announced that the company will participate in the 21st Annual Needham Virtual Healthcare Conference, taking place April 11-14, 2022.

Presentation Details:
Conference – 21st Annual Needham Virtual Healthcare Conference
Date / Time – Tuesday, April 12th, 11:00 AM ET

Presenter – Neal Fowler, Chief Executive Officer at Pathalys Pharma
Format: – Virtual presentation

Pathalys will also be participating in one-on-one investor meetings throughout the conference. Investors interested in scheduling a meeting with the Pathalys management team should contact their Needham representative.

 

About Pathalys Pharma, Inc.

Pathalys Pharma, Inc. is a private, late-stage clinical biopharmaceutical company committed to the development of multiple advanced therapeutics that address unmet needs in the management of ESKD. Pathalys’ initial asset is upacicalcet, a novel calcimimetic with the potential to improve the treatment of SHPT in hemodialysis patients. Beyond upacicalcet, Pathalys continues to identify other high priority needs and potential solutions for patients with ESKD. Pathalys is headquartered in Research Triangle Park, North Carolina.

For more information about Pathalys, please visit www.pathalys.com.

 

Contact Information

Media Contact:
Michael Parks
484.356.7105

Investor Contact:
Patrick Treanor
ptreanor@pathalys.com

 

Pathalys Pharma Announces Phase 3 Program for Upacicalcet

Fort Developer on April 7, 2022

– Plans for Phase 3 Program Presented at National Kidney Foundation’s Annual Spring Clinical Meeting –

Research Triangle Park, N.C., April 7, 2022 – Pathalys Pharma, Inc., a private, late-stage biopharma company committed to the development and commercialization of a range of best-in-class therapies for patients with end stage kidney disease (ESKD), today announced plans for a phase 3 program to support the application of upacicalcet (designated PLS240 for clinical trials), globally. Pathalys holds exclusive rights for the development and commercialization of upacicalcet, a novel calcimimetic, worldwide outside of Japan and Asia.  

“This is a very exciting time for Pathalys as we set out to further understand the full clinical potential of upacicalcet in patients with ESKD through two phase 3 studies that will form the basis for our applications for approval,” said Dr. Theodore Danoff, Chief Medical Officer and SVP, Clinical Development at Pathalys. “Upacicalcet is a novel calcimimetic that performed exceptionally well in phase 3 studies in Japan where it was recently approved for the treatment of SHPT in patients on dialysis. We look forward to the opportunity to demonstrate its effectiveness for patients in the US and other markets globally.”

In a poster presented today at the National Kidney Foundation’s Annual Spring Clinical Meeting (SCM22), the company disclosed its plans to initiate two phase 3 clinical trials in 2022 to evaluate upacicalcet as a treatment alternative to currently available calcimimetics.

The two identically designed phase 3 clinical trials will assess the efficacy of upacicalcet in reducing intact parathyroid hormone (iPTH) by 30 percent or more in participants with secondary hyperparathyroidism (SHPT) and currently on hemodialysis.

Each trial will randomize approximately 375 patients (2:1) to upacicalcet or placebo. Eligible patients will be adults receiving in-center hemodialysis three times weekly and whose iPTH values are 450 pg/mL or greater. Upacicalcet will be titrated based on iPTH and serum corrected calcium (cCa) levels with dose steps between 25 and 300 µg as a bolus IV injection given at the end of a dialysis session. Upacicalcet starting dose will be 50 µg if cCa is 9.0 mg/dL or greater or 25 µg if cCa is below 9.0 mg/dL. Doses will be titrated to maintain iPTH in the range of 150-300 pg/mL.

 

Japanese Approval and Clinical Trial:

Upacicalcet was approved last year in Japan (under the brand name UPASITA®) based in part on a randomized, double-blind, placebo-controlled phase 3 study in hemodialysis patients with SHPT. The study evaluated upacicalcet when administered three times weekly with a starting dose of 25 or 50 μg IV based on cCa levels and was titrated from 25 to 300 μg over 24 weeks.

The primary endpoint was the proportion of patients with a mean iPTH between 60 and 240 pg/mL over weeks 22 to 24 and was achieved in 69 of 103 patients (67.0 percent) in the upacicalcet group and 4 of 50 patients (8.0 percent) in the placebo group (difference of 59.0 percent, p<0.001).

A 30 percent or greater reduction in mean iPTH over weeks 22 to 24 was achieved by 80.6 percent in the upacicalcet group compared to 8.0 percent in the placebo group (difference of 72.6 percent; p<0.001).

Upper gastrointestinal disorder adverse drug reactions (ADRs) were reported in two of 103 patients (1.9 percent) in the upacicalcet group and in three of 50 patients (6.0 percent) in the placebo group. The incidence of calcium less than 7.5 mg/dL was low with 2 events (1.9 percent).

 

About Pathalys Pharma, Inc.

Pathalys Pharma, Inc. is a private, late-stage clinical biopharmaceutical company committed to the development of multiple advanced therapeutics that address unmet needs in the management of ESKD. Pathalys’ initial asset is upacicalcet, a novel calcimimetic with the potential to improve the treatment of SHPT in hemodialysis patients. Beyond upacicalcet, Pathalys continues to identify other high priority needs and potential solutions for patients with ESKD. Pathalys is headquartered in Research Triangle Park, North Carolina.

For more information about Pathalys, please visit www.pathalys.com.

 

Contact Information

Media Contact:
Michael Parks
484.356.7105

 

Investor Contact:

Patrick Treanor
ptreanor@pathalys.com