Bottom Line Up Front:

• Thesis: Larimar (LRMR) is a compelling, pure-play story on a first-in-class therapy for Friedreich’s Ataxia (FA) that addresses the root cause of the disease. Seemingly still under the shadow of a now-resolved clinical hold, the company’s current stock price could have significant upside.
• Upcoming Catalysts: The big one is the imminent data from the Open-Label Extension (OLE) study by the end of this month, which will give us the first long-term look at the 50 mg dose. This will very likely be followed by the planned Biologics License Application (BLA) submission in Q2 2026.
• Valuation Anchor: My overly conservative base case, which assumes no price premium to the competition and a similar penetration despite disease-modifying capabilities, still points to a valuation well north of the current stock price ($4.48 as of 9/22/2025 to a conservative $12/share on positive data). The margin of safety here is substantial.
• Most Material Risk: The key near-term risks are the known potential for anaphylaxis and lower-than-expected biomarker levels in the upcoming readout. On the accelerated approval pathway, nomlabofusp needs to demonstrate the ability to increase frataxin — the protein missing in FA patients resulting in disease — to levels reasonably likely to predict clinical benefit. Longer term, the concern would be that the robust biomarker data doesn’t translate into a clear clinical benefit in the eventual confirmatory trial.

Friedreich’s Ataxia: A Disease of Lost Energy

Before diving into Larimar, it’s important to understand the disease the team over there is fighting. FA is a rare, inherited, and relentlessly progressive neurodegenerative disorder. It’s caused by a specific genetic mutation called a repeat expansion — think of it as a sort of genetic “stutter” — in the frataxin gene. It is also important to note this is an autosomal recessive disorder, meaning this so-called genetic stutter is present in both copies of the gene in FA patients. In healthy individuals, this gene provides the instructions for making a vital protein called frataxin. In FA patients, the section of the gene’s code repeated over and over prevents the cell’s machinery from being able to read the instructions correctly to produce healthy protein. The result is a severe shortage of functional frataxin. [3]

So why is this one protein so critical? Frataxin’s place of employment is inside our cellular power plants, the mitochondria. These tiny engines are responsible for generating the energy that fuels every action we take, in the form of adenosine triphosphate (ATP). The mitochondria are especially crucial in high-energy-demand cells, like the neurons in our nervous system and the muscle cells in our heart. ATP is produced via the electron transport chain (ETC), a sort of cellular transmission line.

Within the mitochondria, two rechargeable batteries — NADH and FADH₂ — feed electricity down the ETC to convert the energy into the fuel our cells need, ATP. Along the ETC are four sub-stations (Complex I → II → III → IV), the first three of which have tiny, but essential, metal relay boxes called iron-sulfur clusters (ISCs) which direct the current through the protein complexes before passing it on to the next sub-station. The energy from the passing electrons is harnessed to pump protons into the intermembrane space of the mitochondria where they are then used by ATP synthase in its production of ATP.

Frataxin’s role is as chief engineer of ISC assembly. Working with other proteins that are a part of the ISC machinery, frataxin assembles the ISCs that are then shipped over to build the relay boxes that are critical for ATP production within the mitochondria. Frataxin is something called an allosteric activator — think of it like this: the other proteins still produce a limited amount of ISCs without frataxin, but they’re a lot slower without the chief engineer. [4] Seems even proteins slack off without their supervisor around. A shortage of ISCs means current is prevented from efficiently transferring through the stations. This results in a cascade of events causing further and further damage within the mitochondria and the wider cell. Iron begins accumulating where it should not, the transmission lines are now leaking electrons, reactive oxygen species (ROS) — akin to sparks flying around damaging all the rest of the machinery — wreak havoc on membranes, proteins, and DNA. Without efficient transmission of electricity, ATP production is significantly handicapped.

The inability of this crucial machinery to function explains why the disease is so devastating. Mitochondria power cells throughout our body and the complications for FA patients are just as far reaching. Symptoms typically begin in childhood, often between the ages of 5 and 15. What starts as a loss of coordination (ataxia), muscle weakness, and fatigue gradually escalates into a multi-system assault. Patients face difficulty speaking, vision and hearing loss, scoliosis, diabetes, and life-threatening heart conditions.

The toll on quality of life is profound. The disease is a slow-motion theft of a person’s physical independence. Most patients are confined to a wheelchair within 10 to 15 years of their first symptoms and are lucky to see their 40th birthday. This isn’t just a set of symptoms; it’s a complete upending of a young person’s life and a heavy burden for their families and loved ones, making the search for a meaningful therapy incredibly urgent. [5]

A Tale of Two Mechanisms: Restoring Power vs. Damage Control

To understand why Larimar’s approach is so compelling, let’s look at the mechanism of their drug and compare it to the only option FA patients currently have. The only FDA approved drug for FA, Biogen’s Skyclarys, works more as a sort of cleanup crew. [6] It doesn’t fix the faulty sub-stations or replace the missing relay boxes. Instead, it works to mitigate the harm caused by the electrical “sparks” (ROS) that fly off the broken transmission lines. [7] This is a valuable intervention, but it’s fundamentally a cleanup operation—it doesn’t address the core power failure.

Larimar’s nomlabofusp is designed to be a disease-modifying therapy—it’s meant to get the power station back online.

Here’s how. As established, the core problem in FA is the chief engineer (frataxin) needed to manufacture the iron-sulfur clusters for the relay boxes is missing. This causes two major problems:

1. Power Failure: The cellular power grid fails because the sub-stations can’t transmit electricity without their relay boxes. This leads to a critical shortage of ATP, causing nerve and heart cells to shut down.
2. Collateral Damage: The unused iron piles up, creating a toxic environment, while the broken transmission lines leak electrons, creating the damaging “sparks” that Skyclarys tries to clean up.

Nomlabofusp acts as the foreign work supply needed when the domestic labor force is insufficient to maintain supply of those crucial parts for the relay boxes. Getting through the cell’s version of Customs and Border Protection (the cell membrane) and into the high-security mitochondrial power plant would normally be impossible for a protein of frataxin’s size. However, this is made possible by a shrewd piece of bioengineering: the foreign engineers are traveling with a cell penetrating peptide that acts both as Global Entry pass, whisking it past CBP sans strip search, and a mitochondrial targeting sequence that allows it to locate and enter the high-security mitochondrial power plant. [8] Once inside the mitochondria an enzyme called mitochondrial processing peptidase clips the off the special peptide that allowed it to pass through the cell membrane (I hoped they booked another appointment to reapply for Global Entry before they left) along with a mitochondrial targeting sequence to locate the mitochondria, nomlabofusp is reduced to healthy frataxin ready to produce the ISCs needed for the sub-stations. This restores the transmission lines, gets the power flowing again, and stops the damaging sparks at their source.

Regulatory & Development Path

The regulatory journey here has been a real comeback story. After a death in a NHP toxicology study testing the highest dose levels, in May 2021 the FDA put a clinical hold on the nomlabofusp clinical program. [9] After discussions with the FDA and producing data from prior studies, Larimar proposed a four-week dose exploration study in FA patients — gathering additional data on safety and tolerability with extended dosing — in a complete response in late August/early September 2022.

The FDA partially lifted the hold in September 2022, allowing the Phase 2 trial to start at the 25 mg dose. After seeing that clean data, the agency allowed the 50 mg cohort to proceed. Finally, in May 2024, after reviewing the full, unblinded safety and biomarker data from both cohorts, the FDA removed the hold completely.

The relationship with the FDA has been unequivocally collaborative. The agency selected nomlabofusp for its START pilot program to accelerate development and then provided the written guidance on using skin frataxin as a surrogate endpoint for a BLA submission planned for Q2 2026. The path forward has never been clearer.

On the Fast Track: The surrogate endpoint to carry nomlabofusp to accelerated approval

The FDA’s Accelerated Approval pathway was designed to allow for earlier approval of drugs that treat serious conditions and fill an unmet medical need. For investigational products that meet this standard, a surrogate endpoint — one that is not a measure of clinical benefit but is reasonably likely to predict clinical benefit — could be used to support the accelerated approval of a drug.

The case for the use of frataxin concentrations as a surrogate in FA is particularly strong. The justification is two-fold. On the one hand, there is ample evidence the median age of onset and rate of disease progression is directly tied to frataxin levels. [3]

Remember, FA is an autosomal recessive disorder. People with only one copy of the mutation, known as asymptomatic carriers, have lower frataxin levels than people without any mutations, yet they don’t show any signs of the disease. These heterozygotes remain phenotypically normal with approximately 50% of normal frataxin concentrations. [10] This is the validation for the surrogate endpoint: raising frataxin levels in FA patients to a range seen in these asymptomatic carriers is our goal post.

In written feedback under FDA’s START pilot, the Agency said it is open to considering skin frataxin concentration as a reasonably likely surrogate endpoint for accelerated approval and recommended a safety database with ≥ 30 participants at 6 months and ≥10 at 12 months, with a large majority on the 50 mg dose. The FDA will make a final decision on whether or not frataxin concentration is a reasonably likely surrogate endpoint during BLA review. Larimar now plans a Q2‑2026 BLA submission that includes OLE and adolescent PK data (expected Sept 2025) and to run a global Phase 3 as the confirmatory study. [11]

The Clinical Evidence: The Power You’re Supplying, It’s Electrifying

For anyone following the FA landscape, the journey of nomlabofusp has been a rollercoaster of hope, setbacks, and now, significant breakthroughs. Let’s break down the clinical trial data and understand why there’s so much excitement around this potential therapy.

Phase 1: Sparks of Change

The initial foray into human trials, the Phase 1 SAD/MAD studies (NCT04176991, NCT04519567), was all about safety and seeing if the drug could even move the needle on frataxin levels in the 55 adult FA patients enrolled. The MAD study is where things got really interesting. It evaluated doses up to 100 mg administered for up to 13 days.

• The 25 mg group received a daily injection for the first 4 days, then a dose every third day, for a total of seven doses.
• The 50 mg group got a daily injection for the first 7 days, followed by a dose every other day, for a total of 10 doses.
• The 100 mg group received a daily injection for all 13 days.

The results from these early studies were the first real proof-of-concept in humans. The drug was generally well-tolerated, with the most common side effects being mild and temporary reactions at the injection site. More importantly, daily dosing at 50 mg and 100 mg led to a more than two-fold increase in frataxin levels in peripheral tissues like blood, buccal (cheek swab) cells, and skin. For the first time, it looked like nomlabofusp could deliver on its promise. [10]

Phase 2: Skin in the Game

Following the promising Phase 1 results, a four-week, placebo-controlled Phase 2 study (NCT05579691) was launched to get a better handle on the 25 mg and 50 mg doses. This trial enrolled two groups: one receiving 25 mg (13 patients) and another receiving 50 mg (15 patients). The dosing regimen involved daily subcutaneous injections for 14 days, followed by every-other-day dosing until day 28.

The top-line results, announced in February 2024, were very encouraging. The drug was again well-tolerated, and the study confirmed a clear, dose-dependent increase in frataxin levels in both skin and buccal cells. At day 14, the end of the daily dosing period, here’s what we saw:

• Skin Frataxin Levels: The 25 mg group saw a median increase of +2.81 pg/μg from baseline, while the 50 mg group saw a jump of +5.57 pg/μg. [12]
• Buccal Frataxin Levels: A similar trend was observed, with a median change of +0.56 pg/μg for the 25 mg cohort and +0.72 pg/μg for the 50 mg cohort. The buccal swab results were more variable, which is why the FDA prefers skin biopsies as a more reliable measure. [12]

Previous studies have measured the mean skin frataxin concentration in healthy controls with 2 normal frataxin alleles is 16.35 pg/µg. [13]

And that’s exactly what happened. After just 14 days of treatment, all patients in the 50 mg cohort who had quantifiable baseline levels saw their frataxin concentrations in skin jump to over 33% of the average level found in healthy volunteers, with several even crossing the 50% threshold.

Open-Label Extension (OLE): Is the Charge Holding?

Most patients from the Phase 2 trial rolled into an ongoing Open-Label Extension (OLE) study, which is designed to gather long-term safety and efficacy data. The study started with a 25 mg daily dose, and now all participants are being moved to a 50 mg daily dose.

Early data from the OLE study on the 25 mg dose show an increase of frataxin concentrations as a percentage of healthy controls go from 16% at baseline to 72% after 90 days. [14] Those are percentages of homozygous, healthy volunteers. Meaning anything over 50% would bring them up to levels of the asymptomatic carriers. Now, there are big questions among physicians on how much improvement increasing frataxin will bring after the accumulation of so much damage, especially in older patients. But, even slowing down progression is a big deal in this patient population.

Finally, various clinical outcomes after 90 days on 25 mg nomlabofusp appeared to show very slight trends towards improvement. [1] Now, this is an open-label study. There is an n of 8. And I mean very slight. But even if only slightly, those slight trends were across the board and when taken in context of the bigger picture, may not be much, but they’re not nothing either.

OLE Daily 25 mg Change from Baseline at Day 90
Statistic	mFARS 93-Point Scale	FARS-ADL 36-Point Scale	Modified Fatigue Impact Scale 84-Point Scale	9 Hole Peg Test Dominant Hand Time (Seconds)
	N = 8	N = 8	N = 8	N = 8
Mean (SD)	-0.69 (3.983)	-2.25 (3.082)	-8.6 (12.24)	-17.79 (27.450)
Median (IQR)	-1.17 (-3.8, 1.2)	-2.25 (-3.8, 0.3)	-3.5 (-19, -3)	-9.00 (-32.0, 1.7)
(Min, Max)	(-5.0, 7.0)	(-8.0, 1.5)	(-28, 9)	(-73.5, 9.8)

The modified Friedreich’s Ataxia Rating Scale (mFARS) is a clinician-scored neurological impairment metric. The Friedreich’s Ataxia Rating Scale Activities of Daily Living (FARS-ADL) measures patient reported difficulty with daily tasks. The Modified Fatigue Impact Scale (MFIS) is a patient reported metric for fatigue. The 9-Hole-Peg-Test is a timed physical test to measure dexterity.

Summary of Frataxin Skin Concentrations Across Studies
Study / Cohort	Sample Size	Timepoint	Dose	Dosing Schedule	Median Baseline (pg/µg)	Baseline (% of healthy)	Median Increase from Baseline (pg/µg)	After Treatment (pg/µg)	After Treatment (% of healthy)
Phase 2 (dose-exp, NCT05579691) – 25 mg	n = 9	Day 14	25 mg	Daily ×14, then QOD to Day 28	3.7	23%	2.81	6.51	40%
Phase 2 – 50 mg	n = 10	Day 14	50 mg	Daily ×14, then QOD to Day 28	2.12	13%	5.57	7.69	47%
Open-Label Extension (OLE) – 25 mg daily → 50 mg	n = 14	Day 90	25 mg	Daily	2.41	15%	9.28	11.69	71%

[14], [15]

Competitors on the Grid

The commercial opportunity in FA is well-defined and validated. There are an estimated 5,000 patients in the U.S. and a similar number in Europe. [16] Biogen’s Skyclarys is well on its way proving this blockbuster opportunity, with sales hitting $130 million in Q2 2025 and analyst peak sales estimates north of $1.5 billion. [6], [17] Biogen actually acquired Skyclarys, which was the crown jewel of that purchase, through its purchase of Reata Pharmaceuticals back in 2023 for a whopping $7.3 billion. [6]

Nomlabofusp is positioned to be the best-in-class therapy because it addresses the underlying protein deficiency. PTC Therapeutics’ vatiquinone, which was looking for the second-place spot in FDA approved therapies for FA, recently received a Complete Response Letter that sends them back to the drawing board for another trial. [18] I don’t truly view vatiquinone (assuming it makes it to market) or Skyclarys as true competitors, however, as:

1. I believe nomlabofusp’s disease-modifying mechanism will prove superior and
2. I can see potential for combination therapy with complementary mechanisms.

Any true long-term “threat” would almost certainly have to have disease-modifying characteristics. As a genetic disorder, the obvious solution that comes to mind is gene therapy. In fact, there is one clinical and several pre-clinical gene therapy programs investigating treatments for FA. Lexeo’s LX2006, the only current clinical program, specifically targets FA cardiomyopathy, but that is years behind yet and comes with its own set of challenges and safety concerns.

Another disease-modifying therapy in the pipeline for FA comes from Design Therapeutics. Their investigational treatment, DT-216P2 is a nifty little piece of molecular wax they’ve dubbed GeneTac® molecules that allow RNA polymerase (your biological iPhone with Live Translation features which transcribes your DNA into RNA, so your monolingual cellular protein factories, also known as ribosomes, can understand their instructions) to do its job without getting stuck on repeat mutations. Wax? Yeah, wax. Hear me out. Repeat expansions, the kind of mutations responsible for FA, are sort of like bent teeth along the zipper that is DNA. These bent teeth make it hard for the slider — the RNA polymerase — to travel down the zipper and efficiently transcribe DNA into RNA so that it may be sent off to the ribosomes and translated into protein. These GeneTac® molecules act almost as a molecular wax, lubricating the path and smoothing it out to allow the slider to travel across. Additionally, this wax has a special affinity for transcriptional elongation complexes, a highly skilled team of zipper mechanics, which help smooth out the track, make sure the RNA polymerase doesn’t fall off, and correct any spelling mistakes Siri makes in the translation. Really cool technology. DT-216P2 is currently in an open-label Phase 1/2 MAD study with 12-week data expected in 2026, where they will look at safety, tolerability, and pharmacokinetics of subcutaneous and intravenous administration. Ultimately, DT-216P2 could provide some competition for nomlabofusp as another disease-modifying therapy. It looks like they may have a leg up on dosing frequency as well. [19]

Sponsor	Therapy	Mechanism Category	Specific Action	Phase of Development	Clinical Data Highlights to Date	Regulatory Designations & Approval Pathway
PTC Therapeutics	Vatiquinone	Oxidative Stress & Mitochondrial Dysfunction	Inhibits the 15-lipoxygenase enzyme to reduce oxidative stress and inflammation.	NDA Rejected; Additional Phase 3 likely required	Phase 3 (MOVE-FA): Failed to meet primary endpoint (mFARS). Showed non-significant trends in some patient subgroups.	Orphan Drug (US & EU), Fast Track (US)
University Federico II	Dimethyl Fumarate	Oxidative Stress & Mitochondrial Dysfunction	Activates the Nrf2 pathway to increase the production of antioxidant proteins and combat oxidative stress.	Phase 2 Completed	Phase 2: Showed activation of the Nrf2 pathway (the drug’s target) and some positive trends on biomarkers of oxidative stress. Full efficacy data pending.	N/A for FA, already approved for other indications.
Larimar Therapeutics	Nomlabofusp	Frataxin Restoration	A fusion protein made up of mature human frataxin and a mitochondrial targeting sequence delivers frataxin to the mitochondria	Phase 2 OLE	Phase 2: Showed dose-dependent increases in frataxin levels in skin and buccal cells. Exploratory data suggests stabilization/improvement in mFARS in the 50 mg cohort.	Orphan Drug (US & EU), Fast Track (US), Rare Pediatric Disease (US)
Lexeo Therapeutics	LX2006	Gene & Cell Therapy	AAV-based gene therapy delivering a functional frataxin gene to cardiac cells to address cardiomyopathy.	Phase 1/2	Phase 1/2 (SUNRISE-FA): Early data from low-dose cohort showed it was well-tolerated and increased frataxin protein expression in heart tissue.	Orphan Drug (US & EU), Fast Track (US), Rare Pediatric Disease (US)
Design Therapeutics	DT-216P2 (Gene-Tac)	Frataxin Restoration	Small molecule that binds to the expanded GAA repeat in the frataxin gene to increase its transcription.	Phase 1	Phase 1: Showed dose-dependent increases in frataxin mRNA in healthy volunteers. Further development paused to address injection site reactions.	Orphan Drug (US & EU), Fast Track (US), Rare Pediatric Disease (US)

Wired for Success

Inside the Control Room: Leadership

The management team, led by CEO Dr. Carole Ben-Maimon, has successfully navigated the company through a challenging clinical hold and emerged with a clear path forward. Their execution on the regulatory front has been excellent.

Dr. Carole Ben-Maimon, MD joined Larimar from Deerfield in 2016 and has over 25 years of experience as an alumnus of Barr, Teva, and Impax Labs. Chief Development Officer Dr. Gopi Shankar PhD, MBA, FAAPS joined Larimar in 2023, with 20 years of experience leading the development of novel biologics, from Janssen Research and Development. Rusty Clayton, DO, serving as both Chief Medical Officer and as the Scientific Advisory Board Chair for Larimar, has more than 15 years of executive experience in the pharmaceutical industry. He was previously Chief Medical Officer at Alcresta Therapeutics, a medical device company, where he led clinical development and medical affairs. Chief Financial Officer Michael Celano brings over 40 years of experience leading life sciences companies with 20 years in Chief Operating Officer, CFO, and board member roles at high-growth life sciences companies.

At Full-Charge: Enough Runway to Get Across the Finish Line

With a pro forma cash balance just north of $200 million after a public offering at the end of July 2025, Larimar has a runway into the fourth quarter of 2026. This is a critical point. They’ve kicked the financing-risk can way down the road. The company is funded through its most important near-term catalyst (OLE data) and its most significant milestone (BLA submission). While they will eventually need to raise capital to fund a commercial launch, they will be doing so from a position of strength, likely after the asset has been significantly de-risked and re-valued by the market.

Ownership & Sentiment

The smart money is showing up to the party. The shareholder base is a who’s who of sharp healthcare funds. Deerfield, owning a very sizeable chunk of the company’s 85.5M shares (~30.7M or 36%), and RA Capital (6M shares) are top-tier specialists who know the space inside and out. Around 9.4M of Deerfield’s shares were added in the public offering in July 2025. [20] Additionally, significant buys in Q2 were made by Fidelity, Millenium, Citadel, and Opaleye. With 75% institutional ownership, the concentration is high, but it’s the right kind of concentration, with smart money making large, long-term bets. [21]

Valuation (Bull / Bear)

Here’s where the story gets interesting. Let’s look at this from a ridiculously conservative angle. Why don’t we assume no price premium for a disease-modifying therapy and that nomlabofusp isn’t able to capture a single additional patient more than what Biogen did last quarter. Why? To show that even if things don’t go perfectly, the margin of safety here is huge.

Skyclarys hit $130 million in sales in Q2. If we annualize that, we get to $520 million in annual sales. [17] Larimar’s market cap is sitting at $383 million (as of 9/22/2025). Back of the napkin math, value the company at 2x peak sales: $1.04 billion market cap/85.6 million shares outstanding = $12ish/share. 

Case	Key Assumptions	Value ($/share)
Bear	– Safety or efficacy issues – Cash returned to shareholders	$1.50
Bull (But Bear-ly)	– Around 30% penetration – $370k annual pricing	$12.00

Let’s not forget, this is a pure binary catalyst. It’s the kind of all or nothing play you’ve got to understand the risk going into. That said, the risk/reward doesn’t look too bad as far as binary catalysts in biotech stocks go. When you consider that nomlabofusp should command a premium price, Skyclarys is not currently approved for pediatrics, 30% penetration for a disease-modifying drug in a condition this severe is quite conservative, and 2x peak sales is lacking for a drug with patent protection into 2040.

Catalysts

Date	Event	What to Watch
Sep 2025	OLE Data Readout	Safety/tolerability (any new allergic reactions), durability of frataxin increase at 50 mg, magnitude of frataxin increase
Q2 2026	BLA Submission	Any change to timeline, confirmation of confirmatory trial design

Risks & Variant Views: What Could Blow a Fuse?

No biotech investment is without risk, and in purely binary situations such as this one, it is especially important to understand the downside. The bear case here centers on two things: safety and efficacy.

Let’s talk about the elephant in the room: safety. There have been injection site reactions observed in 100% of patients across all active arms, compared with around half of the placebo arm depending on the study. Anaphylaxis has also been deemed an adverse drug reaction likely related to nomlabofusp by the safety team. [22] This is a real concern, but not one that isn’t a consideration in any clinical studies. The company is proactively managing it by adding an antihistamine premedication regimen to the OLE protocol. Additionally, one patient in the Phase 1 MAD study experienced a non-serious grade 3 (severe) event of increased hepatic enzymes that was considered to be related to the study drug. [10] That patient was on the 25 mg dose of nomlabofusp. The fact that the full clinical hold was lifted after the agency saw the unblinded Phase 2 data suggests the risk is considered manageable.

The bigger question is efficacy. While the biomarker data is fantastic, the definitive link to long-term clinical improvement still needs to be proven in a confirmatory study. If the upcoming OLE data shows frataxin levels waning over time, or if new safety issues emerge, nomlabofusp’s path to accelerated approval may not be so clear. The last piece to consider is early reads on functional outcomes. While not required for accelerated approval, the direction and magnitude of changes in those metrics could have a significant impact on where the stock price goes.

[1]        “Larimar Therapeutics – Corporate Deck (August 2025).” Accessed: Sept. 22, 2025. [Online]. Available: https://investors.larimartx.com/static-files/4e96ebda-9703-44cf-b4a4-8176a2144889

[2]        “Larimar Therapeutics, Inc. (LRMR) Stock Price, News, Quote & History – Yahoo Finance.” Accessed: Sept. 22, 2025. [Online]. Available: https://finance.yahoo.com/quote/LRMR/

[3]        H. L. Plasterer, E. C. Deutsch, M. Belmonte, E. Egan, D. R. Lynch, and J. R. Rusche, “Development of Frataxin Gene Expression Measures for the Evaluation of Experimental Treatments in Friedreich’s Ataxia,” PLoS ONE, vol. 8, no. 5, p. e63958, May 2013, doi: 10.1371/journal.pone.0063958.

[4]        C.-L. Tsai and D. P. Barondeau, “Human frataxin is an allosteric switch that activates the Fe-S cluster biosynthetic complex,” Biochemistry, vol. 49, no. 43, pp. 9132–9139, Nov. 2010, doi: 10.1021/bi1013062.

[5]        A. Y. Tsou et al., “Mortality in Friedreich Ataxia,” J. Neurol. Sci., vol. 307, no. 1–2, pp. 46–49, Aug. 2011, doi: 10.1016/j.jns.2011.05.023.

[6]        K. Dunleavy, “Biogen pays $7.3B to acquire Reata and newly approved Skyclarys.” Accessed: Sept. 21, 2025. [Online]. Available: https://www.fiercepharma.com/pharma/biogen-ponies-73b-rare-disease-specialist-reata-and-potential-blockbuster-skyclarys

[7]        D. R. Lynch et al., “Safety and Efficacy of Omaveloxolone in Friedreich Ataxia ( MOXIe Study),” Ann. Neurol., vol. 89, no. 2, pp. 212–225, Feb. 2021, doi: 10.1002/ana.25934.

[8]        M. G. Baile, J. Jones, N. Sahr, and G. Shankar, “Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Functional Frataxin into Mitochondria,” AAPS J., vol. 27, no. 3, p. 68, Mar. 2025, doi: 10.1208/s12248-025-01054-5.

[9]        “Larimar Therapeutics Reports FDA Clinical Hold on CTI-1601 and Termination of Recently Announced Private Placement Financing – Larimar Therapeutics, Inc.” Accessed: Sept. 21, 2025. [Online]. Available: https://investors.larimartx.com/news-releases/news-release-details/larimar-therapeutics-reports-fda-clinical-hold-cti-1601-and

[10]      R. Clayton et al., “Safety, pharmacokinetics, and pharmacodynamics of nomlabofusp ( CTI ‐1601) in Friedreich’s ataxia,” Ann. Clin. Transl. Neurol., vol. 11, no. 3, pp. 540–553, Mar. 2024, doi: 10.1002/acn3.51971.

[11]      “Larimar Therapeutics Announces FDA Recommendations on Safety Database, and Other Details of Nomlabofusp BLA Submission for Friedreich’s Ataxia Program – Larimar Therapeutics, Inc.” Accessed: Sept. 22, 2025. [Online]. Available: https://investors.larimartx.com/news-releases/news-release-details/larimar-therapeutics-announces-fda-recommendations-safety

[12]      I. C. MPH, “Friedreich Ataxia Therapy Nomlabofusp Increases Frataxin Levels in Phase 2 Study | NeurologyLive – Clinical Neurology News and Neurology Expert Insights.” Accessed: Sept. 22, 2025. [Online]. Available: https://www.neurologylive.com/view/phase-2-trial-data-shows-friedreich-ataxia-therapy-nomlabofusp-increases-frataxin-levels

[13]      R. Clayton, F. De Toni, N. Scherer, E. Schapiro, M. Hamdani, and G. Shankar, “Prediction of tissue frataxin levels with long term administration of nomlabofusp in adults with Friedreich’s ataxia using modeling and simulations,” Genet. Med. Open, vol. 3, p. 102321, 2025, doi: 10.1016/j.gimo.2025.102321.

[14]      “Larimar Therapeutics Announces Positive Initial Data from Ongoing Long-term Open Label Extension Study & Progress Across Nomlabofusp Program for Friedreich’s Ataxia – Larimar Therapeutics, Inc.” Accessed: Sept. 22, 2025. [Online]. Available: https://investors.larimartx.com/news-releases/news-release-details/larimar-therapeutics-announces-positive-initial-data-ongoing

[15]      “Larimar Therapeutics Reports Positive Top-line Data from Phase 2 Dose Exploration Study from 25 mg and 50 mg Cohorts of Nomlabofusp in Patients with Friedreich’s Ataxia – Larimar Therapeutics, Inc.” Accessed: Sept. 24, 2025. [Online]. Available: https://investors.larimartx.com/news-releases/news-release-details/larimar-therapeutics-reports-positive-top-line-data-phase-2-dose

[16]      B. Polek, M. J. Roach, W. T. Andrews, M. Ehling, and S. Salek, “Burden of Friedreich’s Ataxia to the Patients and Healthcare Systems in the United States and Canada,” Front. Pharmacol., vol. 4, 2013, doi: 10.3389/fphar.2013.00066.

[17]      “Biogen – Q2 2025 Press Release (Exhibit 99.1).” Accessed: Sept. 22, 2025. [Online]. Available: https://investors.biogen.com/static-files/55e019f0-7896-4d3f-89b1-8e8a9e300ae5

[18]      “PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA | PTC Therapeutics, Inc.” Accessed: Sept. 22, 2025. [Online]. Available: https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-receives-complete-response-letter-vatiquinone

[19]      “Our Approach – Design Therapeutics.” Accessed: Sept. 22, 2025. [Online]. Available: https://www.designtx.com/our-approach/

[20]      “Form 4 for Deerfield Management Company LP filed 08/04/2025.” Accessed: Sept. 22, 2025. [Online]. Available: https://investors.larimartx.com/static-files/0261fd43-16c0-407a-ac5e-4ebb49efac27

[21]      “LRMR – Larimar Therapeutics, Inc. Stock – Stock Price, Institutional Ownership, Shareholders (NasdaqGM).” Accessed: Sept. 22, 2025. [Online]. Available: https://fintel.io/so/us/lrmr

[22]      “Larimar Therapeutics Provides Nomlabofusp Development Update and Reports Fourth Quarter and Full Year 2024 Financial Results – Larimar Therapeutics, Inc.” Accessed: Sept. 22, 2025. [Online]. Available: https://investors.larimartx.com/news-releases/news-release-details/larimar-therapeutics-provides-nomlabofusp-development-update-and