On March 4, 2024 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the fourth quarter and full year ended December 31, 2023 (Press release, Nektar Therapeutics, MAR 4, 2024, View Source [SID1234640741]).

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Cash and investments in marketable securities at December 31, 2023, were $329.4 million as compared to $505.0 million at December 31, 2022. Nektar’s cash and marketable securities are expected to support strategic development activities and operations into the third quarter of 2026.

"We believe that the progress that we have made in the past nine months puts Nektar in a strong position to advance our highly promising immunology and inflammation pipeline programs," said Howard W. Robin, President and CEO of Nektar. "We are looking forward to multiple potential value-creating data readouts for REZPEG in the first half of 2025 in both atopic dermatitis and alopecia areata. As we build our pipeline in immunology, we are also conducting IND-enabling studies for NKTR-0165, our novel agonist antibody targeting TNFR2."

Summary of Financial Results

Revenue in the fourth quarter of 2023 was $23.9 million as compared to $22.0 million in the fourth quarter of 2022. Revenue for the year ended December 31, 2023 was $90.1 million as compared to $92.1 million in 2022.

Total operating costs and expenses in the fourth quarter of 2023 were $57.4 million as compared to $74.5 million in the fourth quarter of 2022. Total operating costs and expenses for the full year 2023 were $353.8 million as compared to $468.2 million in 2022. Operating costs and expenses for both the fourth quarter and the full year 2023 decreased as compared to 2022 primarily due to decreases in research and development expenses, general and administrative expense and restructuring, impairment and costs of terminated program, partially offset by $76.5 million in non-cash goodwill impairment recorded in the first quarter of 2023.

R&D expense in the fourth quarter of 2023 was $29.9 million as compared to $34.7 million for the fourth quarter of 2022. R&D expense for the year ended December 31, 2023 was $114.2 million as compared to $218.3 million in 2022. R&D expense decreased for full year 2023 primarily due to the wind down of the bempegaldesleukin program.

G&A expense was $17.3 million in the fourth quarter of 2023 and $21.9 million in the fourth quarter of 2022. G&A expense for the full year 2023 was $77.4 million as compared to $92.3 million in 2022. G&A expense decreased for the full year 2023 primarily due to the wind down of the bempegaldesleukin program.

Restructuring, impairment and other costs of the terminated program were $2.9 million in the fourth quarter of 2023 and $52.0 million in the full year 2023, as compared to $11.6 million in the fourth quarter of 2022 and $135.9 million in the full year 2022. The full year 2023 amount includes $7.9 million in severance expense, $35.3 million in non-cash lease impairment charges, $5.5 million for clinical trial and related employee compensation costs for the wind down of the bempegaldesleukin program, and $3.3 million in other restructuring costs. The full year 2022 amount includes $30.9 million in severance expense, $65.8 million in non-cash lease impairment charges, $31.7 million for clinical trial and related employee compensation costs for the wind down of the bempegaldesleukin program, as well as $7.5 million in other restructuring costs.

Net loss for the fourth quarter of 2023 was $42.1 million or $0.22 basic and diluted loss per share as compared to a net loss of $59.7 million or $0.32 basic and diluted loss per share in the fourth quarter of 2022. Net loss for the year ended December 31, 2023 was $276.1 million or $1.45 basic and diluted loss per share as compared to a net loss of $368.2 million or $1.97 basic and diluted loss per share in 2022. Excluding the $111.8 million in non-cash goodwill and other impairment charges, net loss, on a non-GAAP basis, for the full year 2023 was $164.3 million or $0.86 basic and diluted loss per share.

2023 and Recent Business Highlights

In March 2024, we entered into a securities purchase agreement with TCG Crossover Fund, an institutional accredited investor, to sell securities in a private placement financing for gross proceeds of approximately $30 million, before deducting expenses.
In December 2023, Nektar’s collaborators from the Cairo Laboratory at New York Medical College presented preclinical data on NKTR-255 in combination with obinutuzumab at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. NKTR-255 significantly enhanced the cytotoxicity of expanded Natural Killer (NK) cells when combined with obinutuzumab against rituximab-resistant Burkitt lymphoma (BL) cells in vitro and significantly improved the survival of mice xenografted with Raji-4RH compared to controls.
In October 2023, Nektar initiated a Phase 2b study of rezpegaldesleukin in patients with moderate-to-severe atopic dermatitis. The Company expects initial data from the study in the first half of 2025.
In October 2023, Nektar presented data from the Phase 1b study of rezpegaldesleukin in patients with atopic dermatitis (AD) in an oral session at the 2023 European Academy of Dermatology and Venereology (EADV) Congress. Patients with moderate-to-severe AD that were treated with rezpegaldesleukin showed dose-dependent improvements in Eczema Area and Severity Index (EASI), Validated Investigator Global Assessment (vIGA), Body Surface Area (BSA), and Itch Numeric Rating Scale (NRS) over 12 weeks of treatment compared to placebo, which were sustained post-treatment over an additional 36 weeks.
In September 2023, Nektar announced a clinical study collaboration with AbelZeta Pharma, Inc. (formerly Cellular Biomedicine Group Inc.) to evaluate NKTR-255 in combination with C-TIL051 in advanced non-small cell lung cancer (NSCLC) patients that are relapsed or refractory to anti-PD-1 therapy. Under the collaboration, AbelZeta will add NKTR-255 to its ongoing Phase 1 clinical trial being conducted at Duke Cancer Institute. Enrollment for this trial is ongoing.
In August 2023, Nektar announced promising new and corrected rezpegaldesleukin efficacy data which were previously reported in 2022 and inaccurately calculated by former collaborator Eli Lilly and Company. Nektar regained the full rights to rezpegaldesleukin from Eli Lilly in April 2023.
In April 2023, Nektar announced a strategic reprioritization and cost restructuring plan in order to enable a new focus of its pipeline on immunology and inflammation programs.
Conference Call to Discuss Fourth Quarter and Year-End 2023 Financial Results

Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time, March 4, 2024.

This press release and live audio-only webcast of the conference call can be accessed through a link that is posted on the Home Page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through April 5, 2024.

To access the conference call, please pre-register at Nektar Earnings Call Registration. All registrants will receive dial-in information and a PIN allowing them to access the live call.

On March 4, 2024 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported that it has entered into an agreement to acquire radioisotope production technology firm ARTMS Inc. (ARTMS), its advanced cyclotron-based isotope production platform, manufacturing plant and stockpile of ultra-pure rare metals required for consumable target production (Press release, Telix Pharmaceuticals, MAR 4, 2024, View Source [SID1234640740]).

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ARTMS, based in Burnaby, British Columbia (BC), Canada, is a privately held, venture-backed company, which specialises in the physics, chemistry and materials science of cyclotron-produced radionuclides. A spin-out company from the internationally renowned TRIUMF, Canada’s particle accelerator centre, ARTMS is a commercial-stage company: its technology is used by the major manufacturing networks to optimise production of a range of medical radioisotopes.

The acquisition of ARTMS is expected to further enhance the vertical integration of Telix’s supply chain and manufacturing by providing a greater level of control and security over each of the Company’s diagnostic isotopes.

ARTMS’ core technology platform is based on the QUANTM Irradiation System (QIS), a complete cyclotron-based isotope production system that is designed to support high efficiency and cost-effective production of commercially important medical isotopes including zirconium-89 (89Zr), gallium-68 (68Ga), technetium‐99m (99mTc) and copper-64 (64Cu).

QIS has demonstrated industry-leading production yields and significantly outperforms conventional cyclotron target systems due to the unique intellectual property and target manufacturing processes that underpin it. Additionally, ARTMS’ portfolio of advanced cyclotron technologies will have immediate application and differentiation in the production of future commercially important alpha-emitting, therapeutic isotopes, including actinium-225 (225Ac) and astatine-211 (211At).

The strategic rationale for the acquisition stems from four key areas of commercial synergy between ARTMS and Telix:

Support for the roll-out of Zircaix[1] (TLX250-CDx) kidney cancer imaging. Telix is currently validating multiple production locations of large-scale 89Zr production in the United States (U.S.) using the QIS system and select manufacturing and pharmacy partners. ARTMS’ QIS is a significant enabler of production capacity and supply chain reliability that works by irradiating an yttrium-89 (89Y) target to produce sterile, filtered, purified and dissolved 89Zr that is ready for radiopharmaceutical use. ARTMS possesses a significant stockpile of ultra-pure 89Y to fabricate sufficient cyclotron targets to support this commercial application.

Ultra large-scale production of 68Ga to support Illuccix lifecycle management. Illuccix already supports limited use of cyclotron-produced 68Ga. ARTMS and Telix have been collaborating since 2020[2] to evaluate the potential of higher curie-scale (Ci) production of Illuccix. One of the goals of this acquisition is to enable Telix to deliver further improvement of Illuccix margins and wider distribution through Telix’s unique pharmacy partnership model. ARTMS holds a commercially significant stockpile of zinc-68 (68Zn), which is irradiated to produce 68Ga.

Enhanced supply chain reliability of commercially useful cyclotron-produced diagnostic radionuclides such as 64Cu and 99mTc. Telix will be working with select pharmacy networks and strategic partners to enhance the reliability and routine production of these isotopes following closing of the acquisition. In particular for 64Cu, for which no commercially viable production network currently exists in the U.S., ARTMS has a substantial commercial stockpile of Nickel-64 (64Ni), the essential raw material for 64Cu production, which is in severely limited global supply.

Development of ‘next generation’ cyclotron targets to support the safe and high-yield production of therapeutic radionuclides. These include alpha emitters, such as 225Ac and 211At. This research and development (R&D) activity is highly aligned with Telix’s state-of-the-art research cyclotron footprint at Telix Manufacturing Solutions (TMS) in Belgium. Telix plans to produce clinical trial quantities of 225Ac in-house by end-2024 and will align the ARTMS R&D footprint with its high-energy cyclotron expansion plans for TMS.
As part of the acquisition, Telix will also acquire the benefit of ARTMS’s production facility and clean rooms, located in Burnaby, BC (Canada). Telix expects to continue to operate and expand ARTMS’ R&D and production capabilities at the Burnaby location to support in-house and customer needs, subject to applicable laws and transaction terms.

The acquisition has potential to be financially accretive, add additional revenue, and have a positive impact on gross margins for Illuccix and Zircaix (when commercially available).

Dr. Christian Behrenbruch, Managing Director and Group CEO of Telix said, "ARTMS has been a trailblazer in the field of ‘next generation’ cyclotron-based isotope production systems and demonstrated production efficiency and yields that eclipse comparable systems. It is our hope that by closely aligning this powerful technology with pharmaceutical development, we will transform the cost, market access and utility of diagnostic and therapeutic radiopharmaceuticals. Cyclotron and accelerator-based isotope production has the potential to significantly increase the capacity and lower the cost of commercially important isotopes, serving as an important adjunct to reactor-based production. We are pleased to be able to expand a fruitful collaboration into a deeper partnership."

Doug Gentilcore, Chief Executive Officer of ARTMS added, "Our aim has always been to ensure key isotopes are available on demand to the populations that need them most, and joining forces with Telix is the ideal way to realise this ambition. With Illuccix, Telix has the most used 68Ga-based imaging agent on the U.S. market, and an additional follow-on 89Zr based product candidate – Zircaix. Our long-term goal is therapeutic radionuclides and together with Telix we believe we have the opportunity to deliver these potentially at commercial scale, including through their extensive late-stage product pipeline."

Deal terms and conditions

The purchase price comprises:

US$42.5 million (approximately AU$65.3 million) upfront consideration which is payable in the form of 5,674,636 Telix ordinary shares to be issued at closing[3];
US$15.0 million (approximately AU$23.0 million) upfront consideration payable in cash at closing;
US$24.5 million (approximately AU$37.6 million) in contingent future earn out payments, payable in cash following achievement of certain clinical or commercial milestones; and
cash earn-outs representing low single to low double-digit percentage of net sales of ARTMS products or Telix products prepared using ARTMS products for defined periods depending on the product location where the sale occurs. All earn-outs which have not otherwise expired will terminate on the 10 year anniversary following completion of the ARTMS acquisition.
The cash upfront consideration is subject to customary working capital, debt and transaction expense adjustments. Telix will issue ordinary shares to the stockholders of ARTMS at closing within its Listing Rule 7.1 placement capacity, in part-consideration for the acquisition. The shares will be subject to escrow restrictions.

Closing of the transaction is subject to customary conditions, including regulatory approvals.

On March 4, 2024 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates," or "MTEM"), a clinical-stage biopharmaceutical company focused on the discovery and development of proprietary targeted biologic therapeutics, engineered toxin bodies ("ETBs"), to create novel therapies with potent differentiated mechanisms of action for cancer, reported an update on its programs (Press release, Molecular Templates, MAR 4, 2024, View Source [SID1234640739]).

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Eric Poma, PhD., Chief Executive and Chief Scientific Officer of MTEM, stated, "ETBs represent a new approach to oncology drug development that continue to show unique biology and monotherapy activity in heavily pre-treated patients. We are particularly excited about the monotherapy activity observed with our MT-6402 program in patients with head and neck cancer."

Company Highlights

Continued single agent activity observed with MT-6402 particularly in heavily pre-treated patients with head and neck cancer who had progressed on multiple prior therapies including checkpoint antibodies.
The first dose of 32 mcg/kg has been cleared with no grade 3 or grade 4 drug-related toxicities in the phase I study for MT-8421 targeting CTLA-4-expressing regulatory T-cells ("Tregs") in the tumor microenvironment. Unique pharmacodynamic effects demonstrating potent Treg clearance and IL-2 increases were observed at the first dose level. Enrollment in the second dose cohort (48 mcg/kg) is on-going.
MTEM intends to initiate a study of MT-0169 in CD38+ acute leukemias in collaboration with MD Anderson Cancer Center.
Preclinical activities related to Bristol Myers Squibb collaboration are on-going.
MT-6402 (PD-L1 ETB)

The Part A dose escalation of the phase I study for MT-6402 has been completed with no Grade 4 or Grade 5 drug-related adverse events observed to date.

In the Part A dose escalation, 10 patients with head and neck squamous cell cancer (HNSCC) were treated at doses of 63, 83, or 100 mcg/kg. Two of these patients were not evaluable for the cycle 1 dose-limiting toxicity ("DLT") period or for efficacy because of early progression and came off study after receiving only one or two doses of MT-6402, respectively. Of the remaining eight head and neck cancer patients, the best responses observed were as follows: three had a partial response ("PR") (two unconfirmed) and a fourth patient had evidence of tumor regression. All four patients had progressed on their previous therapies after multiple lines of treatment including checkpoint antibodies. Additional details on each of these participant’s clinical profile and response to the investigational treatment are provided below.

1 patient with a PD-L1 TPS of 2% who had progressed after chemotherapy, radiation therapy, and pembrolizumab had a confirmed PR with 70% tumor reduction and remains on study in cycle 18 (1 cycle = 4 weeks).
1 patient with a PD-L1 CPS of 10% who had progressed after three previous lines of therapy, including progression on Ipi/Nivo within 4 months, showed deepening tumor reduction over time of 3%, 9%, and 15% at the end of cycles 2, 4, and 6, respectively. At the end of cycle 8, the patient had an unconfirmed PR with a 37% reduction in tumor size. The patient remains on study in cycle 9.
1 patient with a PD-L1 CPS of 5% who had progressed after six prior lines of therapy and was refractory to pembrolizumab received 2 doses of MT-6402 before coming off treatment due to the treating physician’s concerns around an asymptomatic grade 1 high sensitivity troponin elevation and hyponatremia related to excessive alcohol intake. The patient discontinued treatment, but a subsequent CT scan assessed by an external radiology review showed that the patient had a 36% tumor reduction (an unconfirmed PR).
1 patient with a PD-L1 CPS of 10% with pre-existing cardiac risk factors of hypertension, hyperlipidemia, and hypercholesterolemia received three doses of MT-6402 before presenting with asymptomatic grade 1 high sensitivity troponin elevation and dosing was held. A CT scan showed a 13% reduction in tumor size, but disease progression occurred during treatment interruption and patient discontinued at the end of cycle 6.
The three other HNSCC patients enrolled in the Part A dose escalation had stable disease of 6, 4, and 2 months, respectively, before disease progression or study discontinuation. One patient progressed at the end of cycle 2. Of these 8 patients, only one patient (the patient with stable disease through 6 cycles) had a PD-L1 tumor proportion score ("TPS") greater than 50%.

"We are very excited to see objective responses in heavily pre-treated, checkpoint-experienced, head and neck cancer patients, a setting with high unmet medical need," said Eric Poma. "Current checkpoint monotherapy in I/O-naïve head and neck cancer patients has a ~15% response rate. Here, in patients who have progressed on checkpoint therapy, we believe we are seeing preliminary evidence of monotherapy activity of long duration and in patients refractory to checkpoint therapy. The partial responses observed to date were in patients with low PD-L1 expression and also showed concomitant increases in cytokines associated with T-cell activation that are not seen with other checkpoint therapies. We believe these data demonstrate a new and potentially best-in-class approach to targeting the PD-1-PD-L1 axis."

"MT-6402 appears generally well-tolerated at the 63 and 83 mcg/kg doses with no Grade 4 or Grade 5 adverse events and no instances of CLS seen at any dose," said Dr. Maurizio Voi, Chief Medical Officer of Molecular Templates. "The irAE profile of MT-6402, including the asymptomatic high sensitivity troponin elevations, appears to be consistent with that seen with other checkpoint therapies." The Part B dose expansion portion of the phase I study in patients with high PD-L1 is ongoing.

MT-8421 (CTLA-4 ETB)

MT-8421, along with MT-6402, represent our unique approach to immuno-oncology based on dismantling the TME through the elimination of immunosuppressive cells in the TME.
MT-8421 is designed to potently destroy CTLA4+ Tregs via enzymatic ribosome destruction but does not have activity against low CTLA-4 expressing peripheral Tregs.
Three patients were dosed in the first cohort of the phase I study at 32 mcg/kg. No grade 3 or grade 4 drug-related adverse events were observed. Two patients have stable disease and remain on study at cycle 4 and cycle 2, respectively (1 cycle = 4 weeks). One patient had disease progression at the end of cycle 2.

The two patients in stable disease showed peripheral depletion of Tregs and significant elevations in IL-2 while on therapy.

Enrollment is on-going in the second cohort of 48 mcg/kg for the phase I study of MT-8421.

MT-0169 (CD38 ETB)

MT-0169 is designed to destroy CD38+ tumor cells through internalization of CD38 and cell destruction via a novel mechanism of action (enzymatic ribosomal destruction and immunogenic cell death).
A phase 1 study in patients with relapsed or refractory multiple myeloma was closed on Dec 2023 due to slow patient enrollment in the wake of multiple new approvals in myeloma. This study enrolled 14 patients and no drug-related Grade 4 or 5 adverse events have been observed. One patient with IgA myeloma who was quad-refractory was treated at 5 mcg/kg and had a stringent Complete Response for 16 cycles (1 cycle = 4 weeks) before discontinuing treatment for progression of disease.
MTEM plans on initiating an investigator sponsored trial with MD Anderson Cancer Center to evaluate MT-0169 in relapsed or refractory CD38+ AML patients.
Research and Collaboration

MTEM continues to make progress in the drug discovery collaboration with Bristol Myers Squibb.
Previously Announced Process to Explore Strategic Alternatives

Previously, Molecular Templates announced that it has retained Stifel, Nicolaus & Company to assist Molecular Templates in initiating a comprehensive evaluation of strategic alternatives, including, but not limited to, potential financing/recapitalization opportunities, the sale of all, or part, of the company, or a merger, or other strategic transactions. A timetable for completion of this strategic review process has not been set, and there can be no assurance that this strategic review will result in any completed transaction.

On March 4, 2024 Urogen Pharma presented its corporate presentation (Presentation, UroGen Pharma, MAR 4, 2024, View Source [SID1234640738]).

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On March 4, 2024 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported that the Company has initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for mirdametinib, an investigational MEK inhibitor, in pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) (Press release, SpringWorks Therapeutics, MAR 4, 2024, View Source [SID1234640737]).

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"There is tremendous potential for mirdametinib to address the substantial needs that exist for children and adults with NF1-PN, and the initiation of our rolling NDA submission brings us one step closer toward our goal of providing these patients with a best-in-class therapy that could make a significant impact on their lives," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are excited to advance the regulatory filing for our second product and look forward to working closely with the FDA on their review of our application."

The NDA submission includes data from the Phase 2b ReNeu trial, a multi-center, open-label study that opened across 50 sites in the U.S. and enrolled 114 patients across two cohorts (pediatric and adult). The primary endpoint was confirmed objective response rate (ORR), defined as ≥ 20% reduction in target tumor volume as measured by MRI and assessed by Blinded Independent Central Review (BICR). As of the data cutoff date of September 20, 2023, the BICR-confirmed objective response rate was 52% in pediatric patients and 41% in adult patients. Mirdametinib treatment showed deep and durable responses and demonstrated significant improvements in key secondary patient-reported outcome measures. Pediatric and adult patients in the ReNeu trial experienced statistically significant improvements from baseline in pain, quality of life, and physical function, as assessed across multiple patient-reported outcome tools. Mirdametinib was generally well tolerated in the trial, with the majority of adverse events (AEs) being Grade 1 or Grade 2. The most frequently reported AEs were rash, diarrhea, and vomiting in the pediatric cohort and rash, diarrhea, and nausea in the adult cohort.

The FDA and the European Commission have granted Orphan Drug designation for mirdametinib for the treatment of NF1. The FDA has also granted Fast Track designation for the treatment of patients ≥ 2 years of age with NF1-PN that are progressing or causing significant morbidity. In July 2023, the FDA granted mirdametinib Rare Pediatric Disease designation for the treatment of NF1, which provides eligibility for a priority review voucher upon FDA approval. SpringWorks expects to complete the NDA submission in the second quarter of 2024.

About the ReNeu Trial

ReNeu (NCT03962543) is an ongoing, multi-center, open-label Phase 2b trial evaluating the efficacy, safety, and tolerability of mirdametinib in patients two years of age and older with an inoperable NF1-associated PN causing significant morbidity. The study enrolled 114 patients to receive mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose of 4 mg twice daily) without regard to food. Mirdametinib is administered orally in a 3-week on, 1-week off dosing schedule and has a pediatric formulation (dispersible tablet) for patients who cannot swallow a pill. The primary endpoint of the ReNeu trial is confirmed objective response rate defined as ≥ 20% reduction in target tumor volume as measured by MRI and assessed by BICR. Secondary endpoints include safety and tolerability, duration of response, and changes from baseline in patient reported outcomes.

About NF1-PN

Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene, which encodes for neurofibromin, a key suppressor of the MAPK pathway.1,2 NF1 is the most common form of neurofibromatosis, with an estimated global birth incidence of approximately 1 in 2,500 individuals, and approximately 100,000 patients living with NF1 in the United States.3,4 The clinical course of NF1 is heterogeneous and manifests in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment.5 Patients with NF1 have an eight to 15-year mean reduction in their life expectancy compared to the general population.2

NF1 patients have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PN, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment; in rare cases, NF1-PN may be fatal.6,7 Patients with NF1-PN can also experience additional manifestations, including neurocognitive deficits and developmental delays. NF1-PNs are most often diagnosed in the first two decades of life.9 These tumors can be aggressive and are associated with clinically significant morbidities; typically, they grow more rapidly during childhood.10,11

Surgical removal of these tumors is challenging due to the infiltrative tumor growth pattern along nerves and can lead to permanent nerve damage and disfigurement.12 MEK inhibitors have emerged as a validated class of treatment for NF1-PN.13

About Mirdametinib

Mirdametinib is a potent, oral, allosteric small molecule MEK inhibitor in development as a monotherapy treatment for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) and low-grade glioma (LGG), and as a combination therapy for the treatment of several subsets of biomarker-defined metastatic solid tumors. Mirdametinib is an investigational drug for which safety and efficacy have not been established.

Mirdametinib is designed to inhibit MEK1 and MEK2, which occupy pivotal positions in the MAPK pathway. The MAPK pathway is a key signaling network that regulates cell growth and survival and that plays a central role in multiple oncology and rare disease indications when genetically altered.

The U.S. Food and Drug Administration (FDA) and the European Commission granted Orphan Drug designation for mirdametinib for the treatment of NF1, and the FDA granted Fast Track designation for the treatment of patients ≥ 2 years of age with NF1-PN that are progressing or causing significant morbidity.