Eledon Pharmaceuticals Announces Orphan Drug Designation Granted to Tegoprubart for the Prevention of Allograft Rejection in Liver Transplantation

Rhea-AI Summary

Eledon Pharmaceuticals (NASDAQ: ELDN) announced that the FDA granted Orphan Drug designation to tegoprubart for the prevention of allograft rejection in liver transplantation on March 10, 2026. Tegoprubart previously held orphan designations for pancreatic islet transplantation and ALS.

The company cites kidney transplant clinical studies and preclinical liver data supporting further evaluation, and anticipates an investigator-sponsored clinical trial to begin later in 2026.

Positive

• FDA granted Orphan Drug designation for tegoprubart in liver transplantation
• Tegoprubart already has two prior FDA orphan designations (pancreatic islet transplantation and ALS)
• Company anticipates an investigator-sponsored clinical trial initiating later in 2026

Negative

• No completed clinical trial data yet demonstrating efficacy of tegoprubart in liver transplantation
• Planned liver transplant study is investigator-sponsored, so timing and enrollment depend on external parties

Market Context

This announcement adds a new orphan indication for tegoprubart in liver transplantation, complementi...

Analysis

This announcement adds a new orphan indication for tegoprubart in liver transplantation, complementing prior designations in islet cell transplantation and ALS. It reinforces Eledon’s transplant-focused strategy while building on prior kidney data, including the Phase 2 BESTOW trial. Investors may track progress of the anticipated investigator-sponsored liver trial, future regulatory interactions, and how clinical data across organs support the long-term development path for tegoprubart.

Key Figures

US rare disease threshold: 200,000 patients EU rare disease threshold: 5 in 10,000 individuals BESTOW trial size: 127 patients +5 more

8 metrics

US rare disease threshold 200,000 patients Definition of rare disease for Orphan Drug status in U.S.

EU rare disease threshold 5 in 10,000 individuals Definition of rare disease for Orphan Drug status in EU

BESTOW trial size 127 patients Phase 2 BESTOW kidney transplantation trial (tegoprubart vs tacrolimus)

Tegoprubart eGFR 69 mL/min/1.73 m² 12‑month mean eGFR for tegoprubart arm in BESTOW

Tacrolimus eGFR 66 mL/min/1.73 m² 12‑month mean eGFR for tacrolimus arm in BESTOW

Q3 2025 net loss $17.5 million Quarter ended September 30, 2025

Liquid resources $93.4 million Cash and short-term investments at 9/30/2025

Recent financing $57.5 million Completed financing funding operations into <b>2Q 2027</b>

Previous Clinical trial Reports

5 past events · Latest: Nov 06 (Positive)

Same Type Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Nov 06	Phase 2 results	Positive	-49.8%	Reported Phase 2 BESTOW kidney transplant results with favorable efficacy and safety profile.
Jul 17	Data presentation	Positive	-1.3%	Announced upcoming Phase 1b tegoprubart kidney transplant data and liver preclinical poster.
Sep 04	Enrollment complete	Positive	-5.8%	Completed enrollment early in Phase 2 BESTOW kidney transplant trial ahead of schedule.
Jul 29	Enrollment update	Positive	-3.4%	Reported enrolling 80 of ~120 patients in BESTOW kidney transplant study worldwide.
Jun 03	Phase 1b update	Positive	+2.5%	Presented updated Phase 1b kidney transplant data showing stable eGFR and tolerability.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Positive clinical updates on tegoprubart have often coincided with negative short-term price reactions.

Recent Company History

Over the past two years, Eledon has repeatedly highlighted clinical progress for tegoprubart in kidney transplantation, including Phase 1b data, enrollment milestones, and full Phase 2 BESTOW results. These events, dated from June 2024 through November 2025, generally described favorable graft function and safety yet frequently saw the stock trade lower in the following 24 hours. Today’s orphan designation in liver transplantation extends the same clinical-transplant narrative into an additional organ setting.

Key Terms

orphan drug designation, allograft rejection, liver transplantation, pancreatic islet cell transplantation, +4 more

8 terms

orphan drug designation regulatory

"today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to tegoprubart"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

allograft rejection medical

"Orphan Drug designation granted to tegoprubart for the prevention of allograft rejection in liver transplantation"

Allograft rejection is the immune system’s attack on a transplanted tissue or organ from another person, where the recipient’s defenses recognize the graft as foreign and try to destroy it. Investors should care because rejection affects the success of transplant procedures and related therapies, drives demand for immunosuppressive drugs and diagnostics, and is a key clinical or regulatory endpoint that can determine a product’s market potential—think of it as a security team mistakenly ejecting an invited guest, undermining the event.

liver transplantation medical

"for the prevention of allograft rejection in liver transplantation"

A surgical procedure that replaces a failing liver with a healthy one from a donor, restoring the organ’s ability to filter blood, produce essential proteins, and support metabolism. Investors track liver transplantation because it drives demand for hospitals, specialized surgeons, transplant centers, immunosuppressive drugs and medical devices; its volume and outcomes influence revenue, regulatory attention and long‑term treatment costs much like a major long‑term service contract.

pancreatic islet cell transplantation medical

"orphan drug designation from the FDA for the prevention of allograft rejection in pancreatic islet cell transplantation"

Pancreatic islet cell transplantation implants clusters of insulin-producing cells into a patient so those cells can sense blood sugar and make insulin, functioning like transplanting tiny factories that restore the body's natural control of glucose. Investors care because a safe, effective and scalable version could change demand for longstanding diabetes drugs and devices, spawn new manufacturing and service markets, and face significant regulatory, cost and supply challenges that affect commercial viability.

amyotrophic lateral sclerosis medical

"and for the treatment of amyotrophic lateral sclerosis (ALS)."

A progressive disease in which nerve cells that control voluntary muscles gradually fail, leading to loss of movement, speech and eventually breathing — like an electrical wiring system in the body slowly shorting out. It matters to investors because there are few effective treatments, so clinical trial results, regulatory approvals, new therapies or diagnostics can rapidly change patient care, market opportunity and company valuations.

calcineurin inhibitors medical

"while reducing the side effects associated with calcineurin inhibitors, supporting its promise"

Calcineurin inhibitors are a class of drugs that lower immune system activity by blocking a protein (calcineurin) that helps immune cells switch on. They are commonly used to prevent organ-transplant rejection and to treat some autoimmune conditions; news about clinical trial results, regulatory approvals, safety concerns, or patent changes can directly affect sales prospects and company valuations, like a dimmer switch that controls how strongly the body’s defenses respond.

immunosuppressive therapy medical

"supporting its promise as a novel immunosuppressive therapy across multiple organ transplant settings"

Immunosuppressive therapy reduces or blocks parts of the immune system with drugs or biologic medicines so the body stops attacking transplanted organs or mistakenly attacking its own tissues. For investors, it matters because these treatments shape clinical trial needs, safety and infection risks, long-term patient monitoring and demand, and regulatory scrutiny—factors that strongly influence a drug’s commercial success and liability profile.

investigator sponsored trial technical

"through an anticipated investigator sponsored trial initiating later this year."

A researcher‑led clinical trial is a study initiated and run by an independent clinician or academic team rather than the company that makes the drug or device; the company may provide the product, data access, or funding but does not control the study. Investors care because these independent trials can provide extra validation, new uses, or safety information that affects a product’s market value—think of an outside expert testing a product in their own lab and reporting unbiased results.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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IRVINE, Calif., March 10, 2026 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to tegoprubart for the prevention of allograft rejection in liver transplantation. Tegoprubart has previously received orphan drug designation from the FDA for the prevention of allograft rejection in pancreatic islet cell transplantation and for the treatment of amyotrophic lateral sclerosis (ALS).

“Clinical studies in kidney transplantation have demonstrated that tegoprubart has the potential to improve graft survival and function while reducing the side effects associated with calcineurin inhibitors, supporting its promise as a novel immunosuppressive therapy across multiple organ transplant settings,” said David-Alexandre C. Gros, MD, Chief Executive Officer of Eledon. “Based on the encouraging preclinical evidence we have generated to date, we believe liver transplantation represents a significant incremental opportunity for tegoprubart, and we look forward to evaluating its potential in the clinical setting through an anticipated investigator sponsored trial initiating later this year.”

Orphan Drug Designation is intended to support the development of therapies for rare diseases, defined as conditions affecting fewer than 200,000 people in the United States or fewer than 5 in 10,000 individuals in the European Union. These designations provide sponsors with a range of incentives intended to encourage the development of medicines for diseases with high unmet medical needs.

About Eledon Pharmaceuticals and tegoprubart

Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for the CD40 Ligand, a well-validated biological target that has broad therapeutic potential. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft transplantation, xenotransplantation, islet cell transplantation, liver transplantation and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com.

Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. Any statements about the company’s planned clinical trials, the development of product candidates, expected or future results of tegoprubart trials and its ability to prevent rejection in connection with liver transplantation, as well as other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “estimates,” “intends,” “predicts,” “projects,” “targets,” “looks forward,” “could,” “may,” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are inherently uncertain and are subject to numerous risks and uncertainties, including: risks relating to the safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sites, as well as patient enrollment; and risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the company’s actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter

Investor Contact:

Stephen Jasper
Gilmartin Group
(858) 525 2047
stephen@gilmartinir.com

Media Contact:

Jenna Urban
CG Life
(212) 253 8881
jurban@cglife.com

Source: Eledon Pharmaceuticals

FAQ

What does the FDA Orphan Drug designation for tegoprubart mean for Eledon (ELDN)?

It means tegoprubart is recognized as a potential therapy for a rare liver transplant indication with development incentives. According to the company, the designation provides regulatory and development incentives intended to encourage therapy development for rare conditions.

When was the orphan designation for tegoprubart in liver transplantation announced by Eledon (ELDN)?

Eledon announced the FDA orphan designation for tegoprubart on March 10, 2026. According to the company, the designation complements prior orphan statuses for pancreatic islet transplantation and ALS, broadening tegoprubart's orphan portfolio.

What clinical evidence supports tegoprubart for liver transplant patients (ELDN)?

There are no completed liver transplant clinical trials yet; support is preclinical and from kidney transplant studies. According to the company, kidney transplant clinical studies showed potential graft benefits and reduced calcineurin inhibitor side effects, guiding liver evaluation plans.

When will Eledon (ELDN) begin clinical trials of tegoprubart in liver transplantation?

The company anticipates an investigator-sponsored trial to start later in 2026, though exact timing is subject to investigator timelines. According to the company, the trial initiation depends on investigator arrangements and regulatory/operational steps.

How does the orphan designation affect tegoprubart's development path for liver transplantation (ELDN)?

Orphan designation may offer incentives like regulatory support and development benefits for tegoprubart in a rare indication. According to the company, these incentives are intended to encourage progress toward clinical evaluation and potential approval for rare-disease uses.