Utah-based Lipocine Inc. announced a strategic pivot to focus exclusively on developing oral treatments for central nervous system disorders, leveraging its proprietary Lip'ral drug delivery platform to advance a pipeline of neuroactive steroids. The biopharmaceutical company revealed the strategic realignment on September 26, 2022, positioning itself to address multiple neuropsychiatric conditions through oral delivery of previously difficult-to-administer molecules.
Targeting CNS Through Neuroactive Steroids
Lipocine's new strategy centers on endogenous neuroactive steroids (NAS), which have demonstrated the ability to impact CNS function through allosteric modulation of the GABAA receptor. According to the company, this novel drug class has received considerable attention for its potential to treat various neuropsychiatric conditions including depression, movement disorders, epilepsy, anxiety, and neurodegenerative diseases.
"We are excited to announce a new strategic direction for Lipocine, which involves applying our validated Lip'ral technology to develop new treatments for CNS disorders," said Dr. Mahesh Patel, President and CEO of Lipocine Inc. "Our initial focus is on endogenous neuroactive steroids which have broad applicability in treating various CNS conditions and where we can leverage our platform to develop highly differentiated oral therapeutics."
The company believes its technology can enable effective oral delivery of endogenous GABAA receptor modulators, which have historically been challenging to deliver orally due to poor aqueous solubility.
Lead Candidate Shows Promise for Postpartum Depression
Lipocine's most advanced NAS candidate is LPCN 1154, a non-invasive oral formulation of the neurosteroid brexanolone being developed for postpartum depression (PPD). The company recently received FDA agreement for establishing efficacy through a pivotal pharmacokinetic bridge to the approved IV infusion brexanolone via a 505(b)(2) NDA filing pathway.
Results from a pilot pharmacokinetic bridge study, serving as a prelude to the pivotal study required for NDA filing, are expected in the first quarter of 2023. Patel emphasized that no other company can produce oral NAS medications with the potential to be fast-acting agents for PPD, noting the oral medication's advantages over current intravenous options as it will not separate mothers from their children during treatment.
Expanding CNS Portfolio
Beyond LPCN 1154, Lipocine's CNS development portfolio includes LPCN 2101, which features a novel mechanism of action for women with epilepsy, and additional undisclosed CNS-focused candidates. The company sees significant potential in the CNS space and believes the strategic focus will allow more effective resource utilization for neuroactive steroid development.
Strategic Asset Management
As part of the realignment, Lipocine intends to explore strategic partnerships and other opportunities for non-core assets. These include the recently FDA-approved testosterone replacement therapy Tlando, which was approved in March 2022 and has been licensed by Antares Pharma. The company plans to explore ex-U.S. commercialization opportunities for Tlando.
Other non-core assets include LPCN 1148 for decompensated liver cirrhosis, currently in a Phase II proof-of-concept study; LPCN 1144 for treatment of non-cirrhotic NASH; LPCN 1107 for prevention of pre-term birth; and LPCN 1111, a once-daily testosterone replacement therapy.
"We continue to believe in the value of our non-core candidates and have determined that the optimal way to advance these assets will be through partnership," Patel explained. "We believe that this strategy will allow us to diversify risk and can create opportunities for non-dilutive financing."
Financial Position and Timeline
Lipocine reported $37.4 million in unrestricted cash, cash equivalents, and marketable investment securities as of June 30, 2022. The company believes this funding will be sufficient to support operations and capital expenditure until at least September 30, 2023. No further significant investment is expected for non-core programs as the company implements its resource-focused strategy.
"The changes we are implementing allow us to focus on a select number of candidates in active development and to manage our resources efficiently," Patel stated, emphasizing the company's commitment to its new CNS-focused direction.
