Approved! FDA Grants IND Clearance to Three More Candidates Today

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The U.S. Food and Drug Administration (FDA) is on a roll granting approvals. Three companies announced Investigational New Drug (IND) application updates today. 

Organicell – COPD Drug 

Miami-based Organicell has been in the news recently with drug candidate Zofin, which saw its IND approved last May for the treatment of COVID-19 patients. The drug is now knee-deep in four different COVID trials, according to the company’s website, after which they plan to pursue an accelerated pathway to a Biologics License Application (BLA) for the drug in COVID patients.  

This week, Organicell announced the approval of another IND application to use Zofin in the treatment of Chronic Obstructive Pulmonary Disease (COPD). An estimated 16 million adults suffer from COPD, with a 2-year mortality rate for severe cases at a mere 50%. 

“COPD is a chronic, degenerative, inflammatory lung disease with no current cure,” said Dr. Mari Mitrani, Chief Science Officer of Organicell. “We are excited to initiate this FDA approved clinical trial and to further explore the potential of Zofin™ in battling this disease.” 

The drug up for approval is a biologic therapeutic derived from perinatal sources, carefully manufactured to maintain the naturally occurring microRNAs without any additives. The next steps will be conducted as a double-blind, placebo-controlled Phase I/II trial of IV infusions of Zofin in COPD patients. Estimated trial initiation is sometime in 2021. 

Passage Bio – Frontotemporal Dementia 

Just seven days after setting the price for its public offering at $22 a pop for a $154 million IPO, Passage Bio announced the FDA has cleared its IND application for PBFT02 gene therapy. The genetic medicines company focuses on rare monogenic CNS disorders.  

The IND approves testing of the therapy in patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. FTD is a form of early onset dementia. Symptoms typically start between ages 40 and 65 but can strike even younger adults. There are currently no approved disease-modifying therapies.  

“FTD can have a devastating impact on a person’s quality of life and create a substantial caregiving and economic burden for families. We are excited to investigate the potential of PBFT02 as a treatment for FTD-GRN as we initiate our clinical development program in the coming months,” said Bruce Goldsmith, Ph.D., CEO of Passage Bio. 

PBFT02 is a single dose delivered via intra-cisterna magna injection. Using an AAV2 viral vector, the therapy delivers a modified DNA to encode the GRN gene to patient’s cells. Providing higher than normal levels of PGRN should help overcome the deficiency in GRN mutation carriers.  

The FDA has already granted Orphan Drug designation to PBFT02 for treatment of FTD-GRN. The hope is to have the therapy in the clinic by the first half of 2021 for a Phase I/II trial.  

KemPharm – Stimulant Use Disorder 

Having filed an IND just before the holidays, KemPharm received the green light from the FDA to begin clinical trials of candidate KP879 to treat stimulant use disorder. The program is anticipated to initiate in 2021.  

KemPharm is focused on prodrugs to treat serious medical conditions through proprietary LAT (ligand activated therapy) technology. The company seeks to generate better prodrug versions of drugs the FDA has already approved, as well as expand to new disease indications.  

The pipeline currently focused on the high-need areas like ADHD and stimulant use disorder. The company licensed Apadaz, the first branded prodrug-based alternative to hydrocodone in 2018. 

Another candidate, KP922, has also been submitted for IND approval for treatment of ADHD. KemPharm already has two other candidates in the clinic for ADHD. Milestone announcements are anticipated in 2021. 

The company relisted on the Nasdaq to begin trading again on January 8 of this year.  

“The successful completion of this series of transactions, culminating in aggregate gross proceeds of approximately $94 million, has allowed the Company to regain its listing on The Nasdaq Capital Market, created the opportunity to eliminate all of the Company’s debt, and has provided a substantial amount of new capital to propel the Company’s efforts to create shareholder value,” said LaDuane Clifton, KemPharm’s Chief Financial Officer. “We are now positioned with a solid balance sheet and a significantly extended cash runway that provides greater operating flexibility as we look forward to the KP415 PDUFA date on March 2, 2021." 

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