Details for Patent: 9,687,519

Which drugs does patent 9,687,519 protect, and when does it expire?

Patent 9,687,519 protects FORZINITY and is included in one NDA.

This patent has thirty-six patent family members in nine countries.

Summary for Patent: 9,687,519

Title:	Methods and compositions for the prevention or treatment of barth syndrome
Abstract:	The disclosure provides methods of preventing or treating Barth Syndrome in a mammalian subject, reducing risk factors associated with Barth Syndrome, and/or reducing the likelihood or severity of Barth Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of TAZ1 in subjects in need thereof.
Inventor(s):	D. Travis Wilson, Mark Bamberger
Assignee:	Stealth Biotherapeutics Inc
Application Number:	US14/771,408
Patent Claim Types: see list of patent claims	Use; Delivery;

United States Patent 9,687,519 (Barth Syndrome): What Claims Actually Cover, How Broad the Method Scope Is, and the US Patent Landscape

Executive summary

US Patent 9,687,519 claims a method of stabilizing cardiolipin remodeling in a mammalian subject with or suspected of Barth Syndrome by administering the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH2 (or pharmaceutically acceptable salts), with an express linkage to decreased TAZ1 expression. Dependent claims narrow to 18:2 cardiolipin species, human subjects, broad routes of administration, combination with a cardiovascular agent, and specific acetate/trifluoroacetate salts. The claim set is method-focused, not composition-of-matter focused, and it is anchored to a biomarker state (TAZ1 downregulation) that can influence infringement analysis, licensing position, and generic or competitor entry risk for Barth Syndrome cardiolipin remodeling therapies.

What does US 9,687,519 claim for stabilizing cardiolipin remodeling in Barth Syndrome?

Core answer: The patent claims a biomarker-linked therapeutic method using a specific peptide, where infringement turns on (i) treating a qualifying subject condition (Barth Syndrome or suspected Barth Syndrome), and (ii) administering a therapeutically effective amount of the specified peptide (or salts), and (iii) a defined biological state: decreased TAZ1 expression versus a normal control.

Claim 1: Required elements and their practical meaning

Claim 1 recites a method for stabilizing cardiolipin remodeling in a mammalian subject having or suspected of having Barth Syndrome comprising administering a therapeutically effective amount of:

• the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH2 (or a pharmaceutically acceptable salt), and
• where the subject has decreased expression of TAZ1 compared to a normal control.

This is a multi-element method claim. The claim scope is defined by three constraints:

1. Therapeutic objective: “stabilizing cardiolipin remodeling”

   • The claim is not framed as “treating Barth Syndrome” generically; it is framed as altering a cardiolipin remodeling process. In practice, infringement and enforcement often require showing the claimed method produces the claimed stabilization effect in the treated subject (or that the administered therapy is used in a way that yields the stabilization).

2. Indication/eligibility: “having or suspected of having Barth Syndrome”

   • This is broader than “diagnosed Barth Syndrome.” “Suspected” can lower evidentiary friction for method infringement, depending on how “suspected” is operationalized in clinical practice (for example, genetic suspicion, biochemical suspicion, or physician suspicion based on clinical findings).

3. Biomarker anchor: “decreased expression of TAZ1 compared to a normal control subject”

   • This is the principal narrowing feature.
   • It also creates a biological-state requirement that is not purely demographic (human vs nonhuman), route, or formulation. It is tethered to a gene-expression metric.

How the TAZ1 language changes the infringement risk calculus

By requiring “decreased expression of TAZ1,” the claim is closer to a patient-selection or diagnostic-defined population method. That can affect:

• whether a competitor’s intended patient selection matches the claimed state,
• whether labeling or clinical protocols restrict use to TAZ1-downregulated subjects,
• how product launch claims (for marketing, reimbursement, or clinical use) should be drafted to reduce exposure.

If a competitor treats Barth Syndrome patients and those patients have TAZ1 downregulation as a matter of biology, the biomarker limitation may be satisfied in a typical Barth Syndrome population. If there exist patient subgroups whose TAZ1 expression does not meet the “decreased compared to normal control” threshold, then the biomarker element can narrow the reachable infringement population.

How to interpret “therapeutically effective amount” in claim scope

“Therapeutically effective amount” is standard functional language. It tends to preserve breadth because it does not pin the dose range in the claim text. For patent-landscape decisions, the key point is that the claim is not limited to a dose or schedule, so infringement exposure can extend across multiple dosing regimens, as long as the administered amount is “therapeutically effective” and the other claim elements are met.

What dependent claims narrow US 9,687,519 and where are the sharp edges?

Core answer: The dependent claims narrow along cardiolipin species, subject identity, administration routes, combination use, and salt identity.

Claim 2: 18:2 species of cardiolipin

Claim 2 limits Claim 1 to where the cardiolipin is 18:2 species of cardiolipin.

Effect on scope

• This is a meaningful narrowing because cardiolipin exists in multiple species by acyl chain composition. Claim 2 confines the method to 18:2 cardiolipin species remodeling stabilization.
• If the claimed therapeutic effect is assessed across cardiolipin species, Claim 1 could still capture the broader “cardiolipin remodeling” stabilization. Claim 2 narrows specifically to 18:2.

Enforcement implication

• A competitor that argues its therapy primarily stabilizes other cardiolipin species could attempt to position outside Claim 2, while still facing Claim 1 exposure if “cardiolipin remodeling” as a process is stabilized broadly.

Claim 3: Subject is human

Claim 3 limits Claim 1 to human subjects.

Effect on scope

• Claim 1 covers “mammalian subject,” so it likely includes nonhuman mammals.
• Claim 3 creates a human-only narrowing dependent claim. In licensing and litigation, this is typically used to broaden meaningful coverage for the actual commercial population (humans) while leaving nonhuman coverage in Claim 1.

Claim 4: Broad route-of-administration coverage

Claim 4 states administration can be oral, topical, systemic, intravenous, subcutaneous, intraperitoneal, or intramuscular.

Effect on scope

• Claim 4 is unusually explicit and covers most standard delivery routes, making route design-around less effective.
• Since Claim 4 is dependent, Claim 1 already likely covers “administering” generically (unless the specification defines route). The dependent claim makes clear the patent owner expects broad route coverage.

Design-around takeaway

• Competitors have limited room to reduce infringement by changing delivery route.

Claim 5: Combination with a cardiovascular agent

Claim 5 adds that the method can further comprise separately, sequentially or simultaneously administering a cardiovascular agent.

Effect on scope

• This claim is drafted to protect combination regimens.
• It covers sequencing and simultaneity. So design-around via “sequential only” or “concurrent only” is not a clean escape.

Commercial positioning implication

• If the peptide therapy is used alongside supportive cardiovascular meds, this claim supports enforcement even if the competitor markets the peptide as an add-on.

Claim 6: Salt identity

Claim 6 narrows the salt to acetate or trifluoroacetate salts.

Effect on scope

• Claim 1 already allows “a pharmaceutically acceptable salt.” Claim 6 narrows one dependent variation to specific salts.
• Salt limitation usually matters for chemistry and product form, but in practice, if the commercial product uses a different pharmaceutically acceptable salt, Claim 6 may not be directly implicated. Claim 1 may still reach other salts unless the specification or prosecution history constrains “pharmaceutically acceptable” in a narrower way.

Does US 9,687,519 protect the peptide itself or only methods?

Core answer: Based on the claim text provided, the patent is protecting a method of treatment, not a composition-of-matter.

• Each claim is a “method for stabilizing cardiolipin remodeling… comprising administering…” structure.
• There is no explicit claim reciting “a peptide composition comprising…” or “a pharmaceutical composition comprising…” in the excerpted claim set.
• That means enforcement is typically directed at clinical use and prescribing, reimbursement coding, or promotional use tied to method claims, rather than direct sales of the peptide substance per se (though in practice method claims can still be enforced through product labeling, instructions, and induced use theories).

What is the practical claim scope for competitor behavior in the US?

Patient population pathway

For a competitor, the biggest potential escape is not route, because Claim 4 is wide. It is either:

• arguing the patient does not satisfy “decreased expression of TAZ1 compared to normal control,” or
• structuring use as not for “stabilizing cardiolipin remodeling,” or
• avoiding administration of the exact peptide (or its salts) in a way that falls outside “therapeutically effective amount” and the claimed peptide identity.

In practice, for a Barth Syndrome-directed therapy using this exact peptide, TAZ1 downregulation is likely inherent in the target biology, so the biomarker limitation may not offer much safe harbor.

Formulation and salt design-around

• Changing from acetate/trifluoroacetate to another salt may avoid Claim 6 specifically.
• It may not avoid Claim 1, which covers “a pharmaceutically acceptable salt.” Unless prosecution history or specification narrows the “salt” definition, Claim 1 likely remains broad on salt identity.

Combination therapy design-around

Claim 5 provides protection even for combination regimens with cardiovascular agents, including sequential administration. Competitors cannot easily avoid by changing co-therapy scheduling.

What patent landscape questions do US 9,687,519 raise for licensing and enforcement?

Even without the rest of the family data, the claim architecture implies a landscape pattern: if the peptide is proprietary, there will often be at least two layers:

1. Method claims tied to disease biology (here: cardiolipin remodeling and TAZ1 downregulation).
2. Compositions and/or manufacturing claims covering the peptide, stereochemistry, salts, and formulation forms.

Given Claim 1’s specificity to the peptide sequence, US 9,687,519 is likely part of a broader estate. The most likely gaps a competitor would target in diligence are:

• whether other US patents in the family claim peptide composition-of-matter,
• whether any patents claim formulation dosage forms or delivery systems (despite Claim 4’s route breadth),
• whether there are method-of-treatment claims that avoid the “TAZ1 decreased expression” constraint by using alternative eligibility criteria (for example, “Barth Syndrome confirmed by genetic testing” or cardiolipin biomarker measures rather than TAZ1 expression).

How strong is the patent estate for US clinical use, based on the claim features?

Core answer: The claims show breadth on route and combination therapy, narrowing mainly on (i) the peptide identity and (ii) the biomarker state (TAZ1 downregulation), plus dependent specificity on 18:2 cardiolipin species and specific salts.

Strength factors

• Defined peptide identity makes the claim clear and enforceable for the exact peptide.
• Route breadth reduces product-form/administration escape routes.
• Combination allowance blocks common real-world co-therapy design-around strategies.
• Human dependent claim aligns with the likely commercial target.

Constraint factors

• Biomarker limitation (TAZ1 downregulation) can be a litigation lever if clinical evidence and patient-selection criteria are contested.
• 18:2 limitation in Claim 2 allows narrower arguments about species-specific effects, though it does not remove Claim 1 coverage.

Where do Section 271(e) exposure and generic entry risk show up?

Core answer: For a competitor trying to launch a Barth Syndrome therapy that uses the same peptide, the method claim is a direct exposure route even if the competitor’s product is “the same drug” in a composition sense. If the competitor is attempting to market a different peptide or a prodrug/analog, the key question is whether it still falls within the defined peptide identity in the claims.

Practical risk areas for entrants

• Labeling and promotion: if marketing ties product use to stabilizing cardiolipin remodeling and targets Barth Syndrome patients with TAZ1 downregulation, method infringement theories become more viable.
• Clinical protocols: if trials or post-approval protocols define eligibility using TAZ1 expression criteria, that can strengthen causation and induced-use theories.
• Combination therapy: if standard-of-care uses cardiovascular agents, Claim 5 broadens exposure for add-on use.

What FDA regulatory status drives Orange Book or BLA biosimilar-type comparisons?

The claim text alone does not establish whether US 9,687,519 maps to an FDA-approved product, whether the product is an NDA or BLA, or whether any Orange Book listings exist for the peptide and salts.

Because the patent is method-only in the excerpted claims, regulatory exclusivity and listing mechanics depend on the approved product’s patent-ownership strategy and FDA listing decisions. That said, the claim’s explicit peptide sequence would typically be a candidate for inclusion in FDA patent listings when the corresponding product exists.

Key Takeaways

• US Patent 9,687,519 is a method-of-use patent centered on stabilizing cardiolipin remodeling in Barth Syndrome, anchored to TAZ1 downregulation.
• Claim 1 is the broadest: it requires the specific peptide D-Arg-2′6′-Dmt-Lys-Phe-NH2 (or a pharmaceutically acceptable salt) at a therapeutically effective amount, administered to a Barth Syndrome or suspected patient with decreased TAZ1 expression.
• Dependent claims narrow to 18:2 cardiolipin (Claim 2), human subjects (Claim 3), enumerated routes including oral/systemic/IV/SC/IM (Claim 4), addition of a cardiovascular agent with flexible sequencing (Claim 5), and acetate/trifluoroacetate salts (Claim 6).
• The estate strength for enforcement is driven by route breadth and combination protection, while the main narrowing lever is the TAZ1 expression requirement.

FAQs

1) Does US 9,687,519 cover nonhuman mammals with Barth Syndrome?

Claim 1 covers “a mammalian subject,” and only Claim 3 is limited to humans.

2) Can a competitor avoid infringement by changing from acetate to another salt?

Claim 6 is limited to acetate or trifluoroacetate, but Claim 1 covers “pharmaceutically acceptable salt,” so changing salts may not remove coverage for Claim 1.

3) Is route of administration a viable design-around?

Claim 4 is explicit and broad (oral, topical, systemic, IV, SC, intraperitoneal, intramuscular). Route changes are unlikely to avoid coverage under Claim 4, and route is not inherently restricted in Claim 1 as written.

4) How does the “TAZ1 decreased expression” limitation affect patient selection?

It adds a biomarker condition tied to “decreased expression… compared to normal control,” which can become central in litigation over whether the treated population meets the claimed biological state.

5) Does the patent extend to combination therapy with cardiovascular drugs?

Yes. Claim 5 explicitly covers methods that further administer a cardiovascular agent, separately, sequentially, or simultaneously.

References

1. US Patent 9,687,519 (claims excerpt provided by user).

Drugs Protected by US Patent 9,687,519

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Stealth Biotheraps	FORZINITY	elamipretide hydrochloride	SOLUTION;SUBCUTANEOUS	215244-001	Sep 19, 2025		RX	Yes	Yes						METHOD FOR TREATING BARTH SYNDROME IN ADULT AND PEDIATRIC PATIENTS WEIGHING AT LEAST 30KG

International Family Members for US Patent 9,687,519

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Canada	2916880
Canada	2916884
Canada	2916977
China	105407906