TFF Pharmaceuticals Announces Additional Positive Data from the Tacrolimus Inhalation Powder (TFF TAC) Phase 2 Trial Following Oral Presentation at the ISHLT 44th Annual Late Breaking Clinical Science Abstract Sessions
- Positive data from the ongoing Phase 2 study of TFF TAC presented at the ISHLT 44th Annual Meeting
- TFF TAC prevents rejection at 1/6 of the oral tacrolimus dose with lower exposure levels
- Gene expression analysis shows normalization of rejection-related genes in patients on TFF TAC
- 4 out of 4 patients completed the 12-week treatment and chose to continue with TFF TAC
- Plans to open an IND in the US for early post-transplant use of TFF TAC
- None.
Analysis of updated trial data is pivotal in understanding the therapeutic potential and safety profile of TFF TAC, particularly in the context of lung transplant recipients. The positive outcomes in preventing rejection at a reduced dosage compared to the oral form indicate a significant step forward in improving the management of immunosuppression, while potentially reducing the incidence of kidney toxicity—a common side effect in organ transplant recipients due to systemic immunosuppressants.
The utilization of Molecular Microscope Diagnostic System (MMDX) represents a notable advancement in validating the suppression of rejection at the molecular level. This data could herald a paradigm shift in how donor organ rejection is monitored and managed post-transplant. The observation that patients are choosing to continue with TFF TAC in the extension phase of the trial suggests a favorable tolerability profile and patient acceptance of the inhalation delivery system.
From a business perspective, transitioning to late-stage testing and opening an IND in the US indicate that TFF Pharmaceuticals is nearing a critical juncture that could substantially impact its market valuation. The focus on improved efficacy with fewer side effects addresses a key unmet medical need and, if successful, could lead to increased market share and revenue generation for the company.
The implications for TFF Pharmaceuticals in the immunosuppressant market, particularly for organ rejection, are notable. The presentation of positive trial results at a reputable scientific meeting such as ISHLT adds credibility and visibility to TFF Pharmaceuticals within the investment community. This can attract investor attention and could potentially influence stock market performance both in the short and long term.
The announcement to proceed with an IND for further trials in the US is a significant development milestone. It suggests confidence in the regulatory acceptance and commercial viability of the product. The pharmaceutical industry often witnesses increased investor speculation around such milestones, which can lead to increased stock volatility. It is important for investors to discern between speculative trading and genuine long-term value, taking into account the clinical and regulatory risks associated with the drug development process.
If TFF TAC can successfully differentiate itself by offering a safer and more efficacious option, it could capture a sizable portion of the market currently held by oral tacrolimus formulations. However, market penetration will depend on several factors, including competitive landscape, pricing strategy and reimbursement policies.
Tacrolimus is a cornerstone in transplant immunosuppression and the inhalation powder form being developed by TFF Pharmaceuticals—TFF TAC—has important pharmacological implications. The reported reduced systemic exposure to tacrolimus necessary to prevent rejection might translate to an improved side effect profile, particularly regarding nephrotoxicity. This is a substantial consideration in transplant medicine where the balance between efficacy and toxicity is critical.
The method of drug delivery is also of interest. By utilizing the TFF technology, a higher concentration of tacrolimus directly to the lungs could enhance local immunosuppression while minimizing systemic absorption. This targeted approach is innovative and may offer a new avenue for other drugs requiring localized delivery.
Furthermore, the Phase 2 data indicating the normalization of rejection-related genes suggests that TFF TAC is not only clinically effective but also molecularly efficacious in preventing rejection. This dual-level confirmation of efficacy could strengthen the drug's profile and supports further investigation into its use as a standard treatment in lung transplant immunosuppression protocols.
New Gene Expression Analysis from Ongoing Phase 2 Study Provides Strong Biomarker Evidence that Treatment with TFF TAC Results in Normalization of Rejection-Related Genes
TFF TAC Prevented Rejection at 1/6 of Oral Tacrolimus Dose and with 2/3 of Oral Tacrolimus Systemic Trough Exposures
4 out of 4 Patients Who Completed the 12-Week Treatment Have Chosen to Remain on TFF TAC by Proceeding to the Safety Extension Phase
FORT WORTH, Texas, April 15, 2024 (GLOBE NEWSWIRE) -- TFF Pharmaceuticals, Inc. (NASDAQ: TFFP) (“the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative drug products based on its patented Thin Film Freezing (TFF) technology platform, today announced additional positive data from the ongoing Phase 2 study which is evaluating Tacrolimus Inhalation Powder (TFF TAC) for the prevention of organ rejection in lung transplant recipients. These new data were presented by Professor Gregory Snell, the lead Principal Investigator of the TFF TAC Phase 2 study, in an oral presentation at the International Society for Heart and Lung Transplantation (ISHLT) 44th Annual Meeting at a late breaking clinical science abstract session, on April 13, 2024, in Prague, Czech Republic.
“The updated Phase 2 data presented at this year’s ISHLT Meeting support the continued advancement of TFF TAC as a highly differentiated immunosuppressive therapy for the prevention of rejection in lung transplant recipients,” said Dr. Zamaneh Mikhak, Chief Medical Officer of TFF Pharmaceuticals. “Thus far, the data suggest that TFF TAC is providing sufficient immunosuppression to prevent rejection at only
“The monitoring of rejection-related genes in transplant recipients through Molecular Microscope Diagnostic System (MMDX) is an innovative approach commonly used in transplant medicine to assess the status of the transplanted organ, giving actionable data to guide immunosuppressive therapy,” said Professor Gregory Snell, Medical Head of Lung Transplant Service at the Alfred Hospital and Lead Principal Investigator of the Phase 2 TFF TAC trial. “The decreased expression of multiple, rejection-related genes provides compelling evidence at a molecular level that patients who have transitioned from oral tacrolimus onto TFF TAC are not experiencing rejection. Based on these updated Phase 2 data, I am encouraged by the evolving profile of TFF TAC as a potentially new advancement in the field of immunosuppression, and I look forward seeing this program move into late-stage testing.”
TFF TAC - updated Phase 2 data post ISHLT 2024 Meeting
The ongoing Phase 2 trial of TFF TAC is an open-label study in lung transplant patients who require reduced tacrolimus blood levels due to kidney toxicity. Part A of the trial is a 12-week treatment period, and Part B is an optional safety extension period. Trial endpoints include safety, tolerability, kidney function, and acute allograft rejection. In March 2024, the Company announced updated data from the first eight patients (N=8) enrolled in the trial. Today, the Company announced additional data from the trial, including new gene expression data assessing molecular markers of acute rejection, that were presented by Professor Gregory Snell at the ISHLT 2024 Annual Meeting on April 13, 2024.
Updated Efficacy
- Successful transition of eight of eight patients from oral Tacrolimus to TFF TAC
- No evidence of acute rejection:
- No signs and symptoms suggestive of acute rejection
- No use of pulse corticosteroids to treat acute rejection
- No deterioration in spirometry suggestive of acute rejection
- No chest x-ray findings suggestive of acute rejection
- No gene expression evidence of rejection in 4/4 patients with available gene expression data on endobronchial biopsies:
- Rejection-related genes normalized on TFF TAC in 3/3 patients with abnormal expression of rejection-related genes at baseline while on oral tacrolimus
- Rejection-related genes remained normal on TFF TAC in one patient with normal expression of rejection related genes at baseline.
- 4/4 patients who completed Part A chose to remain on TFF TAC and proceeded to Part B.
Updated Safety
- No mortality
- No TFF TAC discontinuation due to an AE
- Majority of TEAEs were Grade 2 or lower in severity
- Maintenance of kidney function
Based on these highly encouraging results, TFF Pharmaceuticals plans to open an IND in the US to explore the use of TFF TAC early post-transplant in preparation for a registrational trial.
ABOUT TFF PHARMACEUTICALS’ THIN FILM FREEZING (TFF) TECHNOLOGY
TFF Pharmaceuticals’ proprietary Thin Film Freezing (TFF) technology allows for the transformation of both existing compounds and new chemical entities into dry powder formulations exhibiting unique characteristics and benefits. The TFF process is a particle engineering process designed to generate dry powder particles with advantageous properties for inhalation, as well as parenteral, nasal, oral, topical and ocular routes of administration. The process can be used to engineer powders for direct delivery to the site of need, circumventing challenges of systemic administration and leading to improved bioavailability, faster onset of action, and improved safety and efficacy. The ability to deliver therapies directly to the target organ, such as the lung, allows TFF powders to be administered at lower doses compared to oral drugs, reducing unwanted toxicities and side effects. Laboratory data suggests the aerodynamic properties of the powders created by TFF can deliver as much as
ABOUT TFF PHARMACEUTICALS
TFF Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company engaging patented rapid freezing technology to develop and transform medicines into potent dry powder formulations for better efficacy, safety, and stability. The company’s versatile TFF technology platform has broad applicability to convert most any drug, including vaccines, small and large molecules, and biologics, into an elegant dry powder highly advantageous for inhalation, or for topical delivery to the eyes, nose and the skin. TFF Pharmaceuticals has two lead drug candidates in the clinic: TFF TAC (Tacrolimus Inhalation Powder) and TFF VORI (Voriconazole Inhalation Powder). The Company continues collaborations with a broad array of pharmaceutical companies, academic institutions, and government partners to revolutionize healthcare around the globe. The TFF Platform is protected by over 170 patents issued or pending in the U.S. and internationally. To learn more about TFF Pharmaceuticals and its product candidates, visit the Company’s website at https://tffpharma.com.
SAFE HARBOR
This press release contains forward-looking statements regarding TFF Pharmaceuticals, Inc., including the advancement of TFF TAC into potentially registration-enabling studies; the expectation that the initial data readouts for TFF TAC will be consistent with the further data from the ongoing Phase 2 clinical trial; and the benefits of the Company’s TFF platform. Those forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause actual results to differ materially. Among those factors are: (i) the risk that the further data from the ongoing Phase 2 clinical trial for TFF TAC will not be favorably consistent with the initial data initial data readouts, (ii) the risk that the Company may not be able to advance to registration-enabling studies for its TFF TAC candidate, (iii) success in early phases of pre-clinical and clinicals trials do not ensure later clinical trials will be successful; (iv) no drug product incorporating the TFF platform has received FDA pre-market approval or otherwise been incorporated into a commercial drug product, (v) the Company has no current agreements or understandings with any large pharmaceutical companies for the development of a drug product incorporating the TFF Platform, (vi) the risk that the Company may not be able to obtain additional working capital with which to continue the Phase 2 clinical trial, or advance to the initiation of registration-enabling studies, for TFF TAC as and when needed and (vii) those other risks disclosed in the section “Risk Factors” included in the Company’s Annual Report on Form 10-K filed with the SEC on March 28, 2024. TFF Pharmaceuticals cautions readers not to place undue reliance on any forward-looking statements. TFF Pharmaceuticals does not undertake, and specifically disclaims, any obligation to update or revise such statements to reflect new circumstances or unanticipated events as they occur, except as required by law.
Investor Relations Contact:
Corey Davis, Ph.D.
LifeSci Advisors
(212) 915-2577
cdavis@lifesciadvisors.com
FAQ
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