Catalyst Pharmaceuticals Announces Strategic Plan for Product Portfolio Expansion for Long-Term Success and Provides Corporate Update

Rhea-AI Summary

Catalyst Pharmaceuticals (CPRX) announced plans to expand its R&D focus, aiming for pipeline growth in earlier stage programs and validated technology platforms. The company reported an estimated $118 million in net revenues for 2020, a 16% increase from 2019, and concluded the year with an estimated $140 million in cash and investments. Catalyst intends to invest more in R&D, seeking a senior executive to oversee this strategy. The development of Firdapse LA is on track, with additional studies planned for HNPP starting in Q1 2021.

Positive

• Estimated Firdapse revenues of approximately $118 million for 2020, reflecting a 16% increase from 2019.
• Ending 2020 with an estimated $140 million in cash and investments, up 48% from 2019.
• Plans to diversify product portfolio beyond amifampridine, targeting earlier stage programs.
• Strategic investment in R&D to enhance drug candidate portfolio.

Negative

• None.

• Company to Expand R&D Focus for Pipeline Growth to include Earlier Stage Programs and Validated Technology Platforms
  
  
• Firdapse® 2020 Net Revenues Estimate of approximately $118 Million; about 16% Increase From 2019
  
  
• Ended 2020 with Estimated $140 Million in Cash and Investments
  
  
• Company Highlights Current Product Development Programs
  

CORAL GABLES, Fla., Jan. 06, 2021 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced an expansion in its strategic priorities related to portfolio planning and research and development. Catalyst also provided preliminary net revenues and cash on hand for fiscal-year 2020, as well as an update on product development activities and current litigation.

“Our board and management team are aligned in the opportunity to recalibrate our strategic priorities for 2021 and for the long-term. We believe that we have the ability to leverage our solid balance sheet and strong cash flow, along with our expertise, in developing and commercializing additional medicines to treat rare and ultra-rare diseases. With this in mind, we intend to move to expand our product portfolio beyond amifampridine, with a focus on validated technology platforms as well as earlier stage programs in other rare disease therapeutic categories,” said Patrick J. McEnany, Catalyst Pharmaceuticals’ Chairman and CEO.

Mr. McEnany continued: “Additionally, I am pleased to report our preliminary estimate indicates that 2020 Firdapse^® net revenues were approximately $118 million, an increase of $16 million from 2019. This reflects strong sales performance in an extremely difficult operating environment because of the COVID-19 pandemic. Lastly, we ended the year with approximately $140 million in cash and investments, which represents an increase of approximately $46 million compared to cash and investments at December 31, 2019, and we continue to have no funded debt.”

Strategic Plan for 2021 and Beyond

Catalyst’s Board of Directors recently approved an expansion in the company's strategic focus to include acquiring or in-licensing innovative, validated technology platforms and earlier stage programs in other therapeutic categories outside of neuromuscular diseases. To accomplish these new priorities, Catalyst is prepared to invest more heavily in research and development, including acquiring earlier stage opportunities and validated innovative technology. Catalyst believes that this strategic expansion better positions the company to build out a broader more diversified portfolio of drug candidates that Catalyst expects will add greater value to the company over the near and long-term.

To spearhead this investment, Catalyst has commenced a national search for a key executive to manage this more progressive strategy. This will likely be an M.D. or Ph.D. with 15-20 years of relevant pharma experience and experience in innovated drug technology. This person will be responsible for portfolio planning and developing medicines from discovery through marketing authorizations, as well as strategic leadership across all R&D activities including, direct oversight of science and clinical research.

Year End 2020 Estimated Financial Results

Firdapse annual net revenues for 2020 are estimated at approximately $118 million, an increase of approximately 16% from 2019. Fiscal-year 2020 was a difficult period for patients and healthcare providers in many ways, including the ability to receive a definitive diagnosis of LEMS and providing access to therapies like Firdapse^®. Catalyst believes that as COVID-19 vaccines are administered over the next few quarters, Catalyst will see a more robust uptake in new patient starts and revenues.

Catalyst also estimates that it ended 2020 with approximately $140 million in cash and investments, compared to $94.5 million at the end of 2019, up $46 million, or approximately 48%.

Year-End 2020 Status of Product Development Programs

Firdapse LA (Long-Acting)

The development of the long-acting formulation of amifampridine phosphate remains on track. A large number of candidate formulations were prepared, and three of the most promising formulations were evaluated in a pharmacokinetic (PK) study completed during the fourth quarter of 2020. The results from this first PK study will be used to inform the design and refinement of future product formulations in 2021, and additional PK work is also expected to be conducted during 2021. Catalyst has also completed a number of advisory board meetings with both patients and doctors in order to establish the optimum target characteristics of Firdapse LA that are desired by the LEMS patient community and treating physicians. Periodic updates on this program will be provided as it proceeds.

HNPP

A proof-of-concept study evaluating Firdapse^® as a treatment for Hereditary Neuropathy with Liability to Pressure Palsies (HNPP) is scheduled to start in the first quarter of 2021. The scientific basis for considering this indication is that leakage of neuron potassium channels is observed in HNPP. Firdapse is a potassium channel blocker and may mitigate the pathological effects of the potassium channel leakage in HNPP patients.

HNPP is an autosomal-dominantly inherited peripheral nerve disease caused by a heterozygous deletion of PMP22 gene, leading to a reduction of PMP22 proteins by 35-50% of normal level, which results in disruption of the myelin sheaths of motor neuron axons. Patients with HNPP often present with focal sensory and motor deficits. The events may be triggered by mild mechanical compressions innocuous to healthy humans. In addition, HNPP patients are also afflicted by fatigue. Catalyst believes that HNPP affects about 6,000 patients in the Unites States.

It is hypothesized that functional demyelination in HNPP results in excessive outward current through internodal voltage-gated potassium channels. Application of a potassium channel blocker, like amifampridine, is expected to improve action potential propagation in HNPP, thereby alleviating sensory/motor deficits and fatigue in HNPP. This hypo

FAQ

What were Catalyst Pharmaceuticals' estimated revenues for 2020?

Catalyst Pharmaceuticals estimated approximately $118 million in revenues for 2020, a 16% increase from 2019.

How much cash did Catalyst Pharmaceuticals have at the end of 2020?

Catalyst Pharmaceuticals ended 2020 with approximately $140 million in cash and investments.

What is Catalyst Pharmaceuticals' plan for R&D in 2021?

Catalyst Pharmaceuticals plans to expand its R&D focus to include earlier stage programs and validated technology platforms.

When will the proof-of-concept study for HNPP start?

The proof-of-concept study evaluating Firdapse as a treatment for HNPP is scheduled to start in the first quarter of 2021.