Rindopepimut (CDX-110)_Dosage, benefits & legal status
Also known as CDX-110, Rintega, PEPvIII-KLH, PF-04948568, PF-04948569, ALT-110, SPX-110, WW-34, anti-EGFRvIII vaccine CDX-110
A peptide cancer vaccine, not a treatment anyone takes. Trial staff injected a short piece of a glioblastoma-only mutant protein into the skin with an immune stimulant; survival was 20.1 against 20.0 months in the phase 3.
Common vial size is what vendors typically sell, not a recommendation. Enter your own vial in the calculator.
What is Rindopepimut (CDX-110)?
Plain language first. The technical label stays, but it never stands alone.Overview
Glioblastoma is a brain cancer that surgery, radiation and temozolomide rarely hold for long, so a vaccine aimed at a mutation present only in the tumor was an appealing idea. In ACT IV, 745 patients were randomized, and the 405 with the least tumor left after chemoradiation were the pre-specified primary group: median survival was 20.1 months with the vaccine against 20.0 months with the control injection (hazard ratio 1.01, p=0.93). Nothing about it was self-administered or purchasable. It was injected into the skin by trial staff, mixed with an immune stimulant, and the only access outside a trial was a compassionate-use program that closed in 2017.
Rindopepimut is the 14-residue synthetic peptide LEEKKGNYVVTDHC, spanning the junction created when the EGFRvIII mutation deletes EGFR exons 2 to 7, joined through an added C-terminal cysteine and a thioether linker to keyhole limpet hemocyanin, the copper-based oxygen-carrying protein of the giant keyhole limpet, whose carrier subunit runs 3,392 residues. Each dose was 500 mcg of that conjugate mixed with 150 mcg of GM-CSF and injected into the skin alongside adjuvant temozolomide, with the aim of raising antibodies and T cells against an epitope that exists in no normal tissue. Celldex Therapeutics carried it to a 745-patient phase 3 (ACT IV), stopped for futility in March 2016, and development was recorded as discontinued that August.
What it's used for
Nothing in medicine. It was an experimental vaccine tested against EGFRvIII-positive glioblastoma in clinical trials, and it was never approved, never marketed and never sold anywhere.
How does Rindopepimut (CDX-110) work?
What it does in the body, and where.A peptide vaccine holds no drug that attacks a tumor: it holds a fragment of a protein, injected so the immune system learns to treat cells carrying that protein as foreign.
- 01The mutation makes a targetEGFRvIII is a version of the epidermal growth factor receptor missing exons 2 to 7. Splicing exon 1 straight onto exon 8 puts a glycine at the seam that appears in no normal protein, so the 13 residues around that seam are a label found on the tumor and nowhere else in the body.
- 02What was actually in the syringeThirteen residues are too small to teach the immune system anything, so a cysteine was added to the end and used to tie the peptide to keyhole limpet hemocyanin, the large oxygen-carrying protein of a marine mollusk that immunologists use because the body reacts strongly to it. About 30 copies of the peptide ride on each carrier subunit. Each dose was 500 mcg of that conjugate mixed with 150 mcg of GM-CSF, a growth factor that pulls antigen-presenting cells into the skin. Nelipepimut-S, the other vaccine on this site, needs no carrier because its nine residues already fit the groove of an HLA molecule.
- 03What the immune system did, and what it did not changeAntibodies against EGFRvIII rose at least fourfold in 85% of patients in the single-arm ACT III, so the shot worked as an immunization. EGFRvIII is patchy inside a tumor and often disappears when the cancer returns, so the immune response did not become longer survival. Cilengitide, the other discontinued glioblastoma peptide on this site, reached the same dead end by a different route, with 26.3 months in both arms of its phase 3.
A schematic of the compound's mechanism, not a diagnostic image.
What else is it used for?
Each group carries its own evidence level.Approved means regulators have cleared it for that purpose. Investigational means it is in trials. Early research means it is being studied and is not established.
Newly diagnosed glioblastoma (ACT IV phase 3)
tested-and-failed- Human trial745 randomized; in the 405 with minimal residual disease, the pre-specified primary population, median overall survival was 20.1 against 20.0 months, hazard ratio 1.01 (95% CI 0.79 to 1.30), p=0.93.
- Human trialAcross everyone randomized, 17.4 against 17.4 months, hazard ratio 0.89, P=.22. Progression-free survival was a listed secondary endpoint and its numbers are not in the published abstract.
- No dataThe control arm received 100 mcg of the carrier protein, not an inert placebo, and the company said that arm outperformed historical expectations. The trial was stopped for futility at a second preplanned interim analysis.
Single-arm phase 2 (ACT III)
Early research- human-early65 patients given the vaccine with maintenance temozolomide: 66% free of progression at 5.5 months from entry, median overall survival 21.8 months, 26% alive at 36 months.
- No dataNo control group, so the comparison was against expectation. The randomized trial built on these numbers found nothing.
Recurrent glioblastoma (ReACT phase 2)
Early research- Human trial73 patients randomized, all receiving bevacizumab 10 mg/kg every 2 weeks: 28% (10 of 36) against 16% (6 of 37) free of progression at 6 months, P=0.12 one-sided against a pre-specified one-sided threshold of 0.2.
- Human trialSurvival hazard ratio 0.53 (95% CI 0.32 to 0.88), P=0.01; responses 30% against 18% (P=0.38); steroids stopped for at least 6 months in 33% (6 of 18) against 0% (0 of 19).
- No dataThe authors asked for validation because the sample was small and response to bevacizumab varies widely in recurrent disease. No confirmatory trial was run.
What the shot did to the immune system
Early research- human-earlyACT III: antibody titers against EGFRvIII rose at least fourfold in 85% of patients, and the rise grew with longer treatment; EGFRvIII was gone from 4 of 6 tumor samples taken after more than 3 months of therapy.
- Human trialReACT: 80% reached titers of 1:12,800 or higher, and those titers tracked with longer survival (hazard ratio 0.17, 95% CI 0.07 to 0.45, P<0.0001).
- No dataA measured immune response is not a clinical result. ACT IV produced the same immunization and no survival difference.
The figure usually quoted, 20.1 months, is not the trial's overall survival: it is the median in the 405 patients with minimal residual disease, the pre-specified primary population. Across all 745 randomized patients the medians were 17.4 against 17.4 months. Summaries often imply a benefit in minimal residual disease, and that has it backward, since minimal residual disease was where the trial looked hardest and the hazard ratio there was 1.01 with p=0.93. Two sets of ACT IV numbers circulate, because the company's March 7, 2016, release gave interim figures of 20.4 against 21.1 months with a hazard ratio of 0.99 while the 2017 publication gives 20.1 against 20.0 and 1.01; the publication is the one to use. The registry status reads Completed rather than Terminated, so the accurate phrasing is stopped early for futility. Databases mislead in two further ways: the 3,392-residue sequence on the FDA substance record is the keyhole limpet hemocyanin carrier rather than the vaccine peptide, and the 2,000.24 Da fragment mass in the same record includes the linker and the carrier's lysine rather than the free 14-mer. Counts differ between registry and paper (82 against 65 patients in ACT III, 127 against 73 in ReACT, 223 sites in 24 countries against 165 hospitals in 22), and ReACT's P=0.12 was one-sided against a pre-specified one-sided threshold of 0.2, not a near miss at 0.05. A retraction linked to ReACT in the registry is the retraction of a journal commentary, not of the trial. Rintega was a proposed name no agency approved, so it was never a marketed brand. Celldex uses many CDX codes, among them CDX-011 glembatumumab vedotin, CDX-1127 varlilumab and CDX-0159 barzolvolimab, so CDX-110 is not interchangeable with its other compounds. No vendor sells it: the drug is a conjugate built by a specific process rather than a catalog peptide, and a search of the market turned up no product listing of any kind.
The published record
Papers and trials about Rindopepimut (CDX-110)
33 papers tagged human · 16 not reviews or lab · 3 clinical trial publications
6 registered trials · none with posted results
Furthest stage reached: Phase 3, completed, no results posted
Counted from Europe PMC indexing, not read by a person; a human tag can land on reviews, commentary or lab papers; clinical trial publications is the narrower count.
At a glance
Four things to know about Rindopepimut (CDX-110)
Evidence
Investigational
Category
Immune support
Common vial
Varies
Half-life
Not established
How much, and do you cycle it?
The range, and whether you take breaks.500 mcg of conjugate with 150 mcg GM-CSF (ACT IV phase 3), monthly, after two priming doses (trial). Two priming doses two weeks apart, then monthly until the tumor progressed Trial dose
ACT IV gave it monthly alongside temozolomide until progression or intolerance. No product exists, so there is no schedule to plan around.
Not establishedNo figure
No pharmacokinetic data were published, and none would be expected for a protein conjugate injected into the skin. The trials measured the immune response instead, as antibody titers against EGFRvIII and skin test reactions.
You can compare this against the whole library to see where it sits.
How is it given?
Dosing context and source notes.Given in hospital from a prepared product, so there is nothing to mix.
Trial dose: 500 mcg per dose. ClinicalTrials.gov, ACT IV (NCT01480479)
Injected into the skin by trial staff, never by a patient. Each 0.8 mL dose in ACT IV held 500 mcg of the peptide-carrier conjugate mixed with 150 mcg of GM-CSF (sargramostim) immediately before use, given twice two weeks apart and then monthly until the tumor progressed, alongside temozolomide 150 to 200 mg/m2 for 5 days of each 28-day cycle. The 500 mcg is the weight of the whole conjugate; how much of it is the peptide itself was never disclosed. The control arm got 100 mcg of the carrier protein alone on the same schedule, so the trial compared peptide plus carrier plus GM-CSF against carrier alone rather than against an inert placebo. There was never a commercial presentation, strength or form, and no adjustment data for kidney or liver problems exist, so the calculator has no dose to draw.
How do you store it?
Before mixing, and after.Given in a hospital or trial clinic, so the pharmacy stores it.
Rindopepimut (CDX-110) is prepared and given by hospital, clinic or trial staff, following the product label or the trial protocol. Some products like this arrive ready to use and others are mixed just before the dose, often with hours rather than weeks of shelf life once prepared. It is not a medicine people store at home.
What are the side effects and cautions?
Known cautions and warnings for this compound.Solubility, computed: 50% charged residues, 29% hydrophobic. Water usually sufficient.
A first-pass rule from peptide manufacturers' guidance. It does not account for disulfide bridges, salt form or how the powder was dried, and it predicts what should be easy rather than what will happen in your vial.
Who should avoid it
- Not studied in anyone allergic to keyhole limpet hemocyanin, GM-CSF, polysorbate 80 or yeast-derived products
- Not studied in anyone who has had a severe allergic reaction to shellfish proteins, since the carrier protein comes from a marine mollusk
- Not studied in anyone with an active infection needing treatment, or taking more than 2 mg a day of dexamethasone
- Not studied in pregnancy or breastfeeding, and no data exist
- Never available outside a clinical trial: nothing was marketed, and the last compassionate-use access was recorded as closed in October 2017
Stop and get help
- Hives, swelling of the face or lips, or trouble breathing after an injection: the carrier protein comes from a marine mollusk, and shellfish anaphylaxis was a trial exclusion
- A new seizure, a sudden change in alertness or new weakness on one side: these occurred in both arms of the phase 3 and are also features of the tumor itself
Common effects
- Grade 3 or 4 low platelet counts in 9% against 6% on the control injection, among the 369 and 372 patients treated in ACT IV
- Grade 3 or 4 fatigue in 2% against 5%, brain swelling in 2% against 3%, seizures in 2% against 2% and headache in 2% against 3%
- Sixteen deaths from adverse events, 9 (4%) in the vaccine arm against 7 (3%) in the control arm; one, a lung clot in a 64-year-old man after 11 months of treatment, was judged potentially related
- In the smaller trials the usual complaint was a sore, red injection site, grade 1 or 2; one phase 2 continued monthly injections for more than 3.5 years and described them as well tolerated
Where this page says nothing is established, that means nobody has studied it, not that a compound is safe.
Is Rindopepimut (CDX-110) approved?
And where the numbers on this page come from.No. Rindopepimut (CDX-110) was never approved, and its development has ended. There is no legal route to obtain it as a medicine.
- Approval statusInvestigational
- RouteInto the skin (intradermal), mixed with GM-CSF
- Checked21 October 2026
- DoseFrom published clinical trials. Not approved by a regulator for this dose.
- Vial sizeFrom what vendors typically list; not a recommendation.
- SourcesEMA, orphan designation EU/3/11/923FDA GSRS substance record, rindopepimut (UNII K3L4X0501F)FDA GSRS record, rindopepimut peptide modification (UNII XE2CRB6S1Q)NCI Thesaurus concept C69076FDA orphan drug designation record 249207Celldex, breakthrough therapy designation (SEC exhibit 99.2)Celldex, data safety monitoring board recommendation (March 7, 2016)ClinicalTrials.gov, ACT IV (NCT01480479)Weller M et al., Lancet Oncology 2017;18:1373-1385 (ACT IV)ASCO Post report of ACT IV (February 10, 2018)Reardon DA et al., Clinical Cancer Research 2020;26:1586-1594 (ReACT)Schuster J et al., Neuro-Oncology 2015;17:854-861 (ACT III)FierceBiotech on the Pfizer agreement (September 2010)AdisInsight drug profile 800020344
What has happened recently
Never approved by any agency and never marketed. Duke University and Johns Hopkins University originated the vaccine; Celldex Therapeutics developed it and obtained FDA orphan designation on November 19, 2007 for EGFRvIII-expressing glioblastoma, a designation the FDA record now shows as withdrawn. Celldex licensed it to Pfizer Vaccines in April 2008, the source of the codes PF-04948568 and PF-04948569; Celldex announced on September 3, 2010 that Pfizer was ending the agreement, calling the program no longer a strategic priority, and worldwide rights returned to Celldex effective November 1, 2010. The European Commission granted orphan designation EU/3/11/923 for glioma on October 27, 2011, to Orphix Consulting GmbH rather than to Celldex, and it was removed from the Union Register at that sponsor's request in November 2020. Celldex announced FDA breakthrough therapy designation on February 23, 2015, on the strength of the ACT III and ReACT phase 2 data. On March 7, 2016, the independent data safety monitoring board recommended stopping ACT IV for futility; Celldex confirmed on May 5, 2016, that it had discontinued the study, development in glioblastoma and in solid tumors was recorded as discontinued on August 8, 2016, and expanded access for recurrent glioblastoma was recorded as no longer available on October 16, 2017. Rintega was Celldex's proposed trade name, used in its 2015 announcements; no agency approved it, so it was never a marketed brand.
You can compare this against the whole library, or read how to check a seller.
What else is like Rindopepimut (CDX-110)?
Others in Immune support, side by side.If you're weighing Rindopepimut (CDX-110), it's worth reading Nelipepimut-S (NeuVax), Tertomotide (GV1001), and Darobactin A in the same class.
A peptide cancer vaccine, not a treatment anyone takes. Trial staff injected a short piece of a glioblastoma-only mutant protein into the skin with an immune stimulant; survival was 20.1 against 20.0 months in the phase 3.
- Dose
- 500 mcg of conjugate with 150 mcg GM-CSF (ACT IV phase 3)
- How often
- Monthly, after two priming doses (trial)
A peptide cancer vaccine given by clinic staff to women with a matching tissue type: nine residues of HER2 mixed with GM-CSF, aimed at cells left behind after breast cancer treatment. The phase 3 found no difference in recurrence.
- Dose
- 1,000 mcg with 250 mcg GM-CSF (PRESENT phase 3)
- How often
- Monthly for 6 months, then boosters every 6 months (trial)
A 16-amino-acid piece of telomerase, injected with GM-CSF to make T cells attack tumors. It is the rare investigational peptide that was approved, in South Korea, and then had the approval taken away.
- Dose
- 0.56 to 1.12 mg per dose (phase 2a trial)
- How often
- Weekly for 4 weeks, then every 2 weeks
Seven amino acids stitched into two fused rings by bacteria that live inside insect-killing nematodes. It blocks the machine that builds the outer wall of Gram-negative bacteria, a target nothing in the clinic hits.
- Dose
- No human dose exists: no person has received it
- How often
- Not applicable
Guides for this compound
- How to Reconstitute a Peptide
Mix peptide powder with bacteriostatic water, step by step.
- How to Read an Insulin Syringe
Read the units, convert to millilitres, and draw the exact dose.
- mcg vs mg: The Peptide Dosing Mistake to Avoid
Your vial says mg and your protocol says mcg. Here is the conversion and how to check it.
- How to Read a Peptide COA
What the purity number does and does not tell you, and how to spot an edited report.
- How to Vet a Peptide Vendor
Independent COAs, HPLC purity, and the red flags that mean walk away.
Sources
- EMA, orphan designation EU/3/11/923(2011)Systematic name giving the full 14-residue sequence and the thioether link to keyhole limpet hemocyanin; EU orphan designation October 27, 2011, sponsor Orphix Consulting GmbH, withdrawn November 2020.
- FDA GSRS substance record, rindopepimut (UNII K3L4X0501F)(2026)The record's 3,392-residue subunit with 23 disulfides and six copper sites is the carrier protein; the peptide appears only as a partial lysine modification averaging 30 copies per subunit.
- FDA GSRS record, rindopepimut peptide modification (UNII XE2CRB6S1Q)(2026)C88H138N22O29S, MW 2000.2369: the peptide plus the thioether linker plus the carrier lysine it attaches to, not the free peptide.
- NCI Thesaurus concept C69076(2026)Synonyms CDX-110 and PF-04948568; CAS 1108208-65-6; defined as an EGFRvIII peptide complexed with hemocyanin from Megathura crenulata, the giant keyhole limpet.
- FDA orphan drug designation record 249207(2007)Designated November 19, 2007, for EGFRvIII-expressing glioblastoma multiforme, sponsor Celldex Therapeutics; shown as withdrawn, and never approved.
- Celldex, breakthrough therapy designation (SEC exhibit 99.2)(2015)FDA breakthrough therapy designation February 23, 2015, for EGFRvIII-positive glioblastoma, granted on the ReACT and ACT III data; ACT IV enrollment completed in December 2014 at 745 patients.
- Celldex, data safety monitoring board recommendation (March 7, 2016)(2016)Board recommended stopping ACT IV; interim figures in minimal residual disease were a hazard ratio of 0.99 with 20.4 against 21.1 months; compassionate-use access offered to patients already treated.
- ClinicalTrials.gov, ACT IV (NCT01480479)(2018)500 mcg of CDX-110 with 150 mcg GM-CSF per 0.8 mL against 100 mcg of carrier protein; schedule, eligibility, 745 enrolled, overall survival as the listed primary outcome measure; no posted results.
- Weller M et al., Lancet Oncology 2017;18:1373-1385 (ACT IV)(2017)745 randomized at 165 hospitals in 22 countries; 405 with minimal residual disease; 20.1 against 20.0 months, hazard ratio 1.01 (95% CI 0.79 to 1.30), p=0.93; grade 3 to 4 and serious event rates; 9 against 7 deaths from adverse events.
- ASCO Post report of ACT IV (February 10, 2018)(2018)Arm sizes 371 and 374; minimal residual disease 195 and 210; across everyone randomized, 17.4 against 17.4 months, hazard ratio 0.89, P=.22.
- Reardon DA et al., Clinical Cancer Research 2020;26:1586-1594 (ReACT)(2020)73 randomized with recurrent disease on bevacizumab: 28% against 16% free of progression at 6 months (P=0.12 one-sided); survival hazard ratio 0.53 (95% CI 0.32 to 0.88), P=0.01; steroids stopped for at least 6 months in 33% against 0%; titers of 1:12,800 or higher in 80%.
- Schuster J et al., Neuro-Oncology 2015;17:854-861 (ACT III)(2015)65 patients, single arm: 66% free of progression at 5.5 months, median survival 21.8 months from entry, 26% alive at 36 months, antibody titers up at least fourfold in 85%, EGFRvIII gone in 4 of 6 later samples.
- FierceBiotech on the Pfizer agreement (September 2010)(2010)April 2008 agreement with Pfizer Vaccines; termination announced September 3, 2010; rights back to Celldex effective November 1, 2010.
- AdisInsight drug profile 800020344(2023)Originators Duke University and Johns Hopkins University; synonyms ALT-110, SPX-110 and Rintega; development discontinued August 8, 2016; expanded access NCT03068650 no longer available from October 16, 2017.
Beyond these, 50 records are indexed. Browse the records