On May 7, 2024 MaaT Pharma, a clinical-stage biotech company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported a business update and highlights its key milestones for 2024 (Press release, MaaT Pharma, MAY 7, 2024, View Source [SID1234642780]).

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Hervé Affagard, CEO and co-founder of MaaT Pharma said: "We’re confidently advancing towards Phase 3 results for MaaT013, meeting milestones and delivering value for shareholders. Recent data at the EBMT annual congress underscores MaaT013’s potential in aGvHD where therapeutic options are insufficient. We are also thrilled to partner with Institut Gustave Roussy (IGR), a world-renowned center in cancer treatment to explore MaaT033’s impact on immune checkpoint inhibitors’ response in non-small cell lung cancer patients, and therefore deepening our development in immuno-oncology. This collaboration further strengthens MaaT Pharma’s leading position in oncology and the medical community’s interest in microbiome drug candidates."

Pipeline highlights

MET-N

MaaT013

In hemato-oncology:
In March 2024, the Company announced the launch of a retrospective multicenter trial called CHRONOS in Europe. Its objective is to provide the Company efficacy data for 3rd-line therapies for patients not receiving MaaT013 or any microbiome intervention. This study was not requested by regulatory authorities and does not impact cash projections as funding has already been secured.
In April 2024, at the 50th annual meeting of the European Society for Blood and Marrow Transplantation (EBMT), the Company presented positive efficacy and safety results in 140 patients treated with MaaT013 in acute graft-versus-host disease (aGvHD) as part of the Early Access Program (EAP). The data presented demonstrates a clear reduction in disease burden and an improved Overall Survival (OS), and highlighted MaaT013’s good safety profile. The results in the 49-patient "ARES-like" subgroup, with the same characteristics as those of the Phase 3 ARES trial (NCT04769895) demonstrated 63% GI-ORR at D28 and Overall Survival (OS) of 49% at one year and 42% at 18 months. This represents a significant increase when compared with the historical data published by Abedin et al. (used by the medical community as the most recent reference) which showed a limited 15% OS at one year in a similar population.
Primary endpoint readout, GI-ORR at D28, of the ARES Phase 3 clinical trial in aGvHD is expected for mid Q4-2024.
In immuno-oncology:
In March 2024, the Company informed on the completion of patient recruitment for the Phase 2a clinical trial PICASSO (NCT04988841) sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, evaluating MaaT013 in combination with Immune Checkpoint Inhibitors (ICI). Topline results expected in Q4 2024/Q1 2025.

MaaT033

In immuno-oncology:
MaaT Pharma announces its participation in the IMMUNOLIFE RHU[1] program, a consortium including academic partners, such as Institut Gustave Roussy (IGR), a world-renowned center in the field of cancer treatment, and biotech companies. IMMUNOLIFE aims to solve the significant problem of primary resistance to immune checkpoint inhibitors (ICI) observed in advanced non-small cell lung cancer (NSCLC) patients following antibiotic uptake. MaaT033, an oral, pooled fecal microbiotherapy, developed by MaaT Pharma will be tested as a concomitant treatment to anti-PD1 therapy to increase the ICI response rate in this Phase 2 randomized multicenter clinical trial including advanced NSCLC patients. Participation in this programwill also grant access to clinical and metagenomic data from a large cohort of cancer patients (bladder, lung and renal) which will be used to improve MaaT Pharma’s artificial intelligence (AI) gutPrint platform. The related costs for MaaT Pharma are limited to clinical product supply in line with previous cash projections.
In neurodegenerative diseases:
In February 2024, the Company announced that the Data Safety and Monitoring Board (DSMB) reviewed safety data in the first 8 patients with Amyotrophic Lateral Sclerosis (ALS) treated with MaaT033 in the Phase 1 clinical trial IASO (NCT05889572). The DSMB, composed of 4 independent experts, including an ALS patient association representative, concluded that safety was good and recommended that the trial continue without modifications.
MaaT Pharma announces the completion of patient recruitment for IASO.

MET-C

MaaT034

In immuno-oncology:
In April 2024, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 in San Diego, California, the Company presented new in vitro data characterizing the metabolites produced by MaaT034 and their impact on immune modulation. MaaT034 may be the first full-co-cultivated ecosystem therapy for immuno-oncology, combining a diverse array of supportive species with functional networks of interest to improve the response to immune checkpoint inhibitors. The results demonstrate that MaaT034 produced key metabolites, recognized as promoting gut barrier restoration and modulating immune responses. This represents a significant advancement in understanding the mechanism of action (MoA) of co-cultured microbiome therapies developed by MaaT Pharma marking a major step towards clinical evaluation.

Corporate update

In March 2024, the Company announced the appointment of Jonathan Chriqui, PharmD, as Chief Business Officer and member of the executive management team. Jonathan will be responsible for MaaT Pharma’s business development and partnering strategies, increasing the Company’s ability to secure partnership deals, in line with its strategic objectives.
MaaT Pharma has engaged in active discussions with prominent US clinicians in the field of stem cell transplantation to explore the most efficient path forward to introduce MaaT013 to patients in the United States. The Company is pleased to announce the production of batches of MaaT013 destined for clinical supply in the US as the Company pursues the readiness phase in advance of clinical study initiation.
The Company believes it has sufficient cash to finance operations to the end of Q3 2024. While the Company does not have sufficient cash to finance its operations for the next twelve months, it has active ongoing discussions to finance operations beyond the end of Q3 2024 and remains confident in extending its cash runway.

Key value creation milestones in 2024

MaaT013
Mid-Q4: Primary endpoint readout (GI-ORR at D28) from the ARES Phase 3 clinical trial in aGvHD
Q4 2024/Q1 2025: Topline results of the PICASSO Phase 2a clinical trial in metastatic melanoma
MaaT033
H2 2024: First DSMB[2] of the PHOEBUS Phase 2b clinical trial in allo-HSCT
H2 2024: Results of the IASO Phase 1 clinical trial in ALS
MaaT034
H1 2024: Candidate selection
H2 2024: Start of the production of the first cGMP batch

Upcoming investor and business conferences participation

June 3-6, 2024 – BIO 2024
June 11-12, 2024 – Portzamparc Mid & Small Caps 2024 Conference
June 25-27, 2024 – Stifel European Healthcare Summit Lyon
July 10-12, 2024 – Microbiome Movement Drug Development Summit

[1] Contract N° ANR-21-RHUS-0017 – RHU stands for Recherche Hospitalo-Universitaire – University Hospital Research

[2] at the recruitment of 60 patients

On May 7, 2024 Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical‑stage T-cell reprogramming company advancing a diverse pipeline of cell therapies for patients with solid tumors, reported that members of its senior management team will participate in the BofA Securities 2024 Health Care Conference on Tuesday, May 14 at 8:40 am PT (Press release, Lyell Immunopharma, MAY 7, 2024, View Source [SID1234642779]).

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A live webcast of the presentation can be accessed through the Investors section of the Company’s website at www.lyell.com. Following the live presentation, a replay of the webcast will be available on the Company’s website following the presentation date.

On May 7, 2024 Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) reported financial results for the three months ended March 31, 2024, and provided an operating forecast and business updates. Ligand management will host a conference call today beginning at 4:30 p.m. Eastern Time to discuss this announcement and answer questions (Press release, Ligand, MAY 7, 2024, View Source [SID1234642778]).

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"We are pleased to report another quarter of strong financial results driven by the performance of our commercial royalty portfolio. Simultaneously, we continue to build our portfolio of development stage royalty assets to deliver future growth," said Todd Davis, CEO of Ligand. "We continue to originate a robust pipeline of royalty opportunities with our proactive business development efforts. This is evidenced by our most recent royalty financing agreement with Agenus which will add several new late stage oncology assets to our portfolio. As we look ahead to the near term, we have several important catalysts in our existing portfolio in 2024. This includes Verona Pharma’s ensifentrine and Merck’s V116, both of which have been assigned PDUFA dates in June, top-line Phase 3 data on Takeda’s soticlestat, expected in the third quarter, and the commercial launch of ZELSUVMI, a much-needed treatment for molluscum contagiosum, in late 2024."

First Quarter 2024 Financial Results
Total revenues and other income for the first quarter of 2024 were $31.0 million, compared with $44.0 million for the same period in 2023. Royalties for the first quarter of 2024 were $19.1 million, compared with $17.6 million for the same period in 2023, with the increase primarily attributable to Amgen’s (Nasdaq: AMGN) Kyprolis, Jazz Pharmaceuticals’ (Nasdaq: JAZZ) RYLAZE, Merck and Co.’s (NYSE: MRK) VAXNEUVANCE and Travere Therapeutics’ (Nasdaq: TVTX) FILSPARI, partially offset by a decline in CASI’s (Nasdaq: CASI) EVOMELA. Captisol sales were $9.2 million for the first quarter of 2024, compared with $10.6 million for the same period in 2023, with the change due to the timing of customer orders. Contract revenue and other income was $2.7 million for the first quarter of 2024, compared with $15.7 million for the same period in 2023, with the difference driven by a $15.3 million milestone payment earned from Travere Therapeutics upon the FDA approval of FILSPARI in the prior year quarter.

Cost of Captisol was $2.9 million for the first quarter of 2024, compared with $3.7 million for the same period in 2023, with the decrease due to lower total Captisol sales. Amortization of intangibles was $8.2 million for the first quarter of 2024, compared with $8.5 million for the same period in 2023. Research and development expense was $6.0 million for the first quarter of 2024, compared with $6.7 million for the same period in 2023. General and administrative expense was $11.0 million for the first quarter of 2024, compared with $10.9 million for the same period in 2023.

Net income from continuing operations for the first quarter of 2024 was $86.1 million, or $4.75 per diluted share, compared with net income from continuing operations of $43.6 million, or $2.43 per diluted share, for the same period in 2023. The increase in net income from the prior year period is due primarily to realized gains from short-term investments associated with Viking Therapeutics (Nasdaq: VKTX) stock of $60.0 million. Adjusted net income from continuing operations for the first quarter of 2024 was $69.7 million, or $3.84 per diluted share, compared to $39.9 million, or $2.28 per diluted share, for the same period in 2023. Excluding the impact of gains from sales of Viking Therapeutics stock, core adjusted net income from continuing operations was $21.8 million, or

$1.20 per diluted share, compared with $23.4 million, or $1.33 per diluted share, for the same period in 2023. The decrease in core adjusted net income is driven by the $15.3 million milestone payment earned from Travere Therapeutics in the prior year quarter. See the table below for a reconciliation of net income from continuing operations to adjusted net income from continuing operations.

As of March 31, 2024, Ligand had cash, cash equivalents and short-term investments of $310.6 million.

2024 Financial Guidance

Ligand is reaffirming its 2024 financial guidance. The Company expects 2024 royalties ranging from $90 million to $95 million, sales of Captisol to range from $25 million to $27 million, and contract revenue ranging from $15 million to $20 million. These revenue components result in total revenue forecast of $130 million to $142 million. Ligand notes that with total revenue of $130 million to $142 million, core adjusted earnings per diluted share are expected to range from approximately $4.25 to $4.75. This guidance excludes the $60 million gain from short-term investments on the sale of Viking Therapeutics stock.

First Quarter and Recent 2024 Business Highlights

On May 7, Ligand announced a $100 million royalty financing agreement with Agenus, Inc. Under the terms of the agreement, in exchange for an initial $75 million payment, Ligand will receive 18.75% of the royalties and 31.875% of the future milestones on six Agenus-partnered oncology programs including BMS-986442 (Bristol Myers Squibb), AGEN2373 (Gilead Sciences), INCAGN2385 and INCAGN2390 (Incyte), MK-4830 (Merck), and UGN-301 (UroGen Pharma). Ligand will also receive a 2.625% royalty on future global net sales of Agenus’ novel immuno-oncology botensilimab in combination with balstilimab ("BOT/BAL") program. Agenus’ BOT/BAL program received Fast Track Designation from the U.S. FDA in April 2023 for patients with metastatic, refractory colorectal cancer that is not MSI-H/dMMR, who do not have liver metastases, and who failed first and second line standard of care treatments. The capital will support Agenus’ upcoming Phase 3 BOT/BAL colorectal cancer trial and other key commercialization activities. Ligand has an option to invest an additional $25 million to increase its economics on a pro rata basis for the additional investment.

On April 3, Ligand announced the creation of Pelthos Therapeutics under the leadership of Scott Plesha as Chief Executive Officer. Pelthos is focused on the commercialization of innovative, safe, and efficacious therapeutic products for patients suffering from conditions with limited treatment options. ZELSUVMI (berdazimer topical gel, 10.3%), its first product, is the first and only FDA-approved prescription medicine for the treatment of the highly transmissible molluscum contagiosum (molluscum) viral skin infection in adults and pediatric patients one year of age and older. It can be applied by patients, parents, or caregivers at home, outside of a physician’s office, or other medical setting. ZELSUVMI received a Novel Drug designation from the U.S. FDA in January 2024 to treat molluscum viral skin infection. ZELSUVMI was developed using Pelthos’ proprietary nitric oxide-based NITRICIL technology platform. Commercial availability of ZELSUVMI in the United States is expected by late 2024. The rights to ZELSUVMI and all assets related to the NITRICIL technology platform were acquired from Novan, Inc. in September 2023.

Portfolio Updates

On April 24, Travere Therapeutics and CSL Vifor (ASX: CSL), Travere’s commercial partner in Europe, gained European Commission conditional marketing authorization (CMA) for FILSPARI (sparsentan) for the treatment of adults with primary IgA nephropathy (IgAN). All member states of the EU, including Iceland, Liechtenstein, and Norway are included in the CMA. The European Commission’s decision follows the Committee for Medicinal Products for Human Use (CHMP)’s positive opinion in February 2024, based on results from the pivotal Phase 3 PROTECT study of FILSPARI in IgAN. The PROTECT study met its primary endpoint at the pre-specified interim analysis with statistical significance.

On May 6, Travere Therapeutics announced the FDA granted Priority Review for its sNDA to convert FILSPARI (sparsentan) from accelerated approval to full approval for the treatment of IgAN in the U.S. with a PDUFA target action date of September 5, 2024.

On April 24, Viking announced that in the first quarter this year they completed the 52-week biopsies for the Phase 2b VOYAGE study of VK2809 in biopsy-confirmed NASH and fibrosis. As we’ve previously mentioned, the study successfully achieved its primary endpoint after 12 weeks of treatment and affirmed VK2809’s potent effect on liver fat, along with its favorable tolerability and safety profile. Viking plans to report data on histologic changes assessed after 52 weeks of treatment later in the second quarter of 2024.

On April 15, Marinus Pharmaceuticals provided an update on the Phase 3 RAISE trial evaluating the safety and efficacy of IV ganaxolone in patients with refractory status epilepticus. The trial did not meet pre-defined stopping criteria at the interim analysis; Marinus has completed RAISE enrollment at approximately 100 patients with topline results expected in the summer of 2024. Future development of IV ganaxolone in refractory status epilepticus will be assessed following review of the final RAISE results. Marinus remains blinded to the RAISE trial data.

Adjusted Financial Measures

Ligand reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, income tax effect of adjusted reconciling items and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, the Company does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, share-based compensation expense and the effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call
Ligand management will host a conference call today beginning at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 715-9871 using the conference ID 8755336. Callers outside the U.S. may dial 1-(646) 307-1963. To participate via live or replay webcast, a link is available at www.ligand.com.

On May 7, 2024 Kyowa Kirin Co reported Consolidated Financial Results Fiscal 2024 First Quarter (Press release, Kyowa Hakko Kirin, MAY 7, 2024, View Source [SID1234642777]).

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On May 7, 2024 – Ionis Pharmaceuticals, Inc., reported financial results for the first quarter ended March 31, 2024 (Press release, Ionis Pharmaceuticals, MAY 7, 2024, View Source [SID1234642776]).

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"Ionis is off to a great start in 2024, as we continue to execute on our vision to bring better futures to people with serious diseases. The WAINUA launch for hereditary ATTR polyneuropathy is progressing well with AstraZeneca. And we are one step closer to our first independent launch with our NDA submission for olezarsen, which is supported by robust data positioning olezarsen to make a profound difference for people with FCS," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "We look forward to presenting positive Phase 3 donidalorsen data, along with data from our open-label extension and ‘switch’ studies in patients with HAE at EAACI later this month, setting the stage for Ionis’ second independent launch. Additionally, we have multiple upcoming data readouts from our mid-stage programs that, if positive, could advance into Phase 3 development, further strengthening our ability to deliver a steady cadence of potentially transformational medicines for years to come."

First Quarter 2024 Summary Financial Results(1):

Three months ended
March 31,

2024

2023

(amounts in millions)

Total revenue

$
119

$
131

Operating expenses

$
269

$
245

Operating expenses on a non-GAAP basis

$
238

$
218

Loss from operations

$
(150
)

$
(114
)
Loss from operations on a non-GAAP basis

$
(119
)

$
(87
)

(1)
Reconciliation of GAAP to non-GAAP basis contained later in this release.

Financial Highlights

•
Revenue for the first quarter of 2024 earned from numerous diverse sources, including a new source of royalty revenue with the launch of WAINUA in the U.S.

•
Continued strategic investments to bring WAINUA, olezarsen and donidalorsen to patients drove increased operating expenses in the first quarter of 2024 compared to the same period last year

•
Cash and short-term investments of $2.2 billion as of March 31, 2024 enable continued investments to drive increasing value, including supporting our planned upcoming launches

1
•
Reaffirmed 2024 financial guidance

Recent Marketed Medicines Highlights

•
WAINUA for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated sales of $5 million in the first partial quarter of launch resulting in royalty revenue of $1 million for Ionis in the first quarter of 2024

•
SPINRAZA for the treatment of spinal muscular atrophy (SMA) generated global sales of $341 million resulting in royalty revenue of $38 million in the first quarter of 2024

o
Biogen presented new positive neurofilament light chain (NfL) biomarker data from the Phase 4 RESPOND study of SMA patients adding further evidence supporting the potential benefit of SPINRAZA in infants and toddlers who had unmet medical needs after treatment with gene therapy

Recent Late-Stage Pipeline Highlights

•
Eplontersen granted Fast Track designation by the FDA for the treatment of patients with ATTR cardiomyopathy

•
Olezarsen achieved multiple milestones advancing it closer to potentially addressing two distinct populations of patients with urgent unmet need, familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG):

o
Submitted NDA to the FDA for FCS

o
Presented positive Phase 3 Balance study data in patients with FCS with a simultaneous publication in the New England Journal of Medicine

o
Presented positive Phase 2b Bridge study data in patients with HTG and sHTG with a simultaneous publication in the New England Journal of Medicine

o
Opened Expanded Access Program (EAP) for FCS in the U.S.

o
Granted Breakthrough Therapy and Orphan Drug designations by the FDA for the treatment of patients with FCS

o
Completed enrollment of the Phase 3 CORE pivotal study and ESSENCE supportive exposure study for sHTG; CORE2 confirmatory pivotal study on track to fully enroll mid-year

•
Donidalorsen achieved multiple milestones advancing it closer to potentially becoming a first-in-class RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE):

o
Reported positive topline data from the Phase 3 OASIS-HAE study in patients treated every four weeks or every eight weeks; preparing to submit NDA

o
Opened EAP for HAE in the U.S.

o
Granted Orphan Drug designation by EMA

•
Bepirovirsen granted Fast Track designation by the FDA for the treatment of patients with chronic hepatitis B (CHB)

Recent Other Pipeline Highlights

•
Reported positive Phase 2 data for ION224 (DGAT2) in patients with metabolic dysfunction-associated steatohepatitis (MASH)

•
Initiated the Phase 1/2 Orbit study of ION356 (PLP1) in patients with Pelizaeus-Merzbacher disease (PMD)

2
First Quarter 2024 Financial Results

"Our first quarter results keep us on track to achieve our 2024 financial guidance. With the launch of WAINUA in the U.S. underway, we are excited to add WAINUA royalties to our meaningful revenues in the first quarter. We believe WAINUA is uniquely positioned in this growing market to address the needs of ATTRv-PN patients who remain significantly underserved, especially as it is the only approved medicine with monthly dosing that can be self-administered via an auto injector," said Elizabeth L. Hougen, chief financial officer of Ionis. "We continued to invest our capital resources in our near-term commercial opportunities, wholly owned pipeline and technology. We expect our modest expense growth this year to be driven by our activities to support the WAINUA launch and planned launches for olezarsen and donidalorsen with R&D expenses approaching steady state as several late-stage studies have recently ended. We believe the investments we are making today and plan to make over the next few years position Ionis to drive increasing value for patients and stakeholders."

Revenue

Ionis’ revenue was comprised of the following:

Three months ended

March 31,

2024

2023

Revenue:

(amounts in millions)

Commercial revenue:

SPINRAZA royalties

$
38

$
50

WAINUA royalties

1

–

Other commercial revenue:

TEGSEDI and WAYLIVRA revenue, net

9

7

Licensing and other royalty revenue

11

11

Total commercial revenue

59

68

Research and development revenue:

Amortization from upfront payments

42

16

Milestone payments

7

23

Collaborative agreement revenue

49

39

WAINUA joint development revenue

11

24

Total research and development revenue

60

63

Total revenue

$
119

$
131

Commercial revenue in the first quarter of 2024 included a new source of royalty revenue with the launch of WAINUA in the U.S. during the first quarter of 2024. While the number of patients on SPINRAZA treatment remained consistent globally, royalties decreased year over year primarily due to the timing of shipments in several markets outside the U.S. Ionis’ commercial revenue in the first quarter of 2024 also included royalties from the net sales of QALSODY, which Biogen launched in the second quarter of 2023.

R&D revenue in the first quarter of 2024 included increased revenue from the amortization of upfront payments compared to the same period last year due to the new collaborations Ionis entered into last year with Roche and Novartis. This increase was offset by decreases in milestone payments due to timing and WAINUA joint development revenue, which decreased as development activities relating to ATTRv-PN wound down with the launch of WAINUA underway.

Operating Expenses

Ionis’ operating expenses increased in the first quarter of 2024 compared to the same period in 2023, consistent with expectations. SG&A expenses increased year over year primarily due to the launch of WAINUA in the U.S. and launch preparation activities for olezarsen and donidalorsen. R&D expenses increased compared to the same period last year due to the timing of development activities and are expected to stabilize in 2024 as several late-stage studies have ended.

3
Balance Sheet

As of March 31, 2024, Ionis’ cash, cash equivalents and short-term investments decreased to $2.2 billion compared to $2.3 billion at December 31, 2023. As the year progresses, the Company plans to continue deploying its capital resources toward growth opportunities. Ionis’ working capital also decreased over the same period primarily due to the Company’s lower cash and short-term investments balance.

Webcast

Management will host a conference call and webcast to discuss Ionis’ first quarter 2024 results at 11:30 a.m. Eastern time on Tuesday, May 7, 2024. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company’s first quarter 2024 earnings slides click here.

For more information about SPINRAZA and QALSODY, visit View Source and View Source, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s).

INDICATION for WAINUA (eplontersen)
WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

IMPORTANT SAFETY INFORMATION for WAINUA (eplontersen)

WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

Please see link to U.S. Full Prescribing Information for WAINUA.