Acoramidis

CDMO Market Report: Key Regulatory Approvals and Clinical Advances in March-April 2025

a month ago

• Multiple CDMOs secured significant contract manufacturing opportunities as regulatory bodies approved new indications for established drugs, particularly in oncology and rare diseases. • AstraZeneca's portfolio saw substantial growth with expanded approvals for Imfinzi, Tagrisso, and Lynparza, strengthening partnerships with contract manufacturers including Lonza, Dottikon, and Samsung Biologics. • Contract manufacturers supporting treatments for autoimmune conditions showed strong performance, with Argenx's Vyvgart Hytrulo receiving expanded indications for myasthenia gravis and CIDP.

Vividion Therapeutics Initiates Phase I Trial of Novel RAS-PI3Kα Inhibitor for Advanced Solid Tumors

2 months ago

• Vividion Therapeutics has dosed the first patient in a Phase I trial of VVD-159642, an oral inhibitor targeting the RAS-PI3Kα pathway implicated in approximately 20% of all cancers. • The study will evaluate VVD-159642 as both monotherapy and in combination with sotorasib or trametinib in patients with advanced solid tumors, potentially offering a new treatment approach for RAS-driven cancers. • VVD-159642 represents Vividion's fourth clinical-stage program from its chemoproteomics platform, designed to selectively inhibit oncogenic signaling while avoiding toxicities that have limited previous attempts to target this pathway.

Alnylam's Vutrisiran Shows Strong Efficacy in ATTR Cardiomyopathy, Setting Stage for New Treatment Standard

3 months ago

• Alnylam's RNAi drug vutrisiran demonstrated a 28% reduction in all-cause mortality and recurrent cardiovascular events in ATTR cardiomyopathy patients, with efficacy increasing to 33% in patients not taking Pfizer's tafamidis. • The HELIOS-B trial showed vutrisiran's benefits increased over time, with a 36% reduction in the primary endpoint at 42 months, positioning the quarterly-administered injection as a potential new standard of care. • Vutrisiran, already approved as Amvuttra for ATTR polyneuropathy, could reach multibillion-dollar sales if approved for cardiomyopathy, though it faces competition from BridgeBio's acoramidis and AstraZeneca/Ionis' eplontersen.

EMA Recommends Approval for New Cancer Treatments and Vaccines

4 months ago

• The European Medicines Agency (EMA) has recommended eight new products for EU-wide approval, expanding treatment options for various conditions. • Several cancer treatments have received positive recommendations, potentially offering new hope for patients with different types of malignancies. • A new antiparasitic combination has been endorsed for use in non-EU markets, addressing a critical need in regions affected by parasitic infections. • The EMA is also reviewing new safety information regarding Leqembi, an Alzheimer's disease treatment, ensuring ongoing monitoring of its benefit-risk profile.

FDA Lifts Clinical Hold on Amylyx's AMX0114 Phase 1 ALS Trial, Paving Way for LUMINA Study

4 months ago

• The FDA has lifted the clinical hold on Amylyx Pharmaceuticals' Phase 1 trial of AMX0114, an antisense oligonucleotide for ALS treatment. • Amylyx is now cleared to initiate screening and enrollment at U.S. sites for the Phase 1 LUMINA trial, expected to begin in Canada in early 2025. • The LUMINA trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in 48 adults with ALS. • AMX0114 targets calpain-2, a protein involved in axonal degeneration, with preclinical data showing improved neuronal survival.

Nuvation Bio's Taletrectinib Gains FDA Priority Review and Expanded Access Program for ROS1+ NSCLC

5 months ago

• The FDA granted priority review to Nuvation Bio's NDA for taletrectinib, a next-generation ROS1 inhibitor, for advanced ROS1+ non-small cell lung cancer, setting a PDUFA date of June 23, 2025. • Taletrectinib demonstrated promising efficacy and tolerability in the TRUST-I and TRUST-II trials, showing durable responses and prolonged progression-free survival in ROS1+ NSCLC patients. • Nuvation Bio has initiated an Expanded Access Program (EAP) in the U.S. for taletrectinib, offering access to eligible patients with advanced ROS1+ NSCLC outside of clinical trials. • The NDA is based on pooled data from the TRUST-I and TRUST-II studies, which included over 300 patients, representing the largest ROS1-positive NSCLC dataset supporting an NDA.

FDA Approves BridgeBio's Attruby, Setting Up Competition with Pfizer's Vyndamax in ATTR-CM Market

6 months ago

• The FDA has approved BridgeBio Pharma's Attruby (acoramidis) for transthyretin amyloidosis cardiomyopathy (ATTR-CM), a heart condition leading to heart failure and death. • Attruby demonstrated a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalizations compared to placebo in the ATTRibute-CM trial. • BridgeBio is launching Attruby at a list price of $18,759 for a 28-day supply, positioning it to compete with Pfizer's Vyndamax, which dominates the ATTR-CM market. • Several other companies, including Alnylam and AstraZeneca/Ionis, are also developing therapies for ATTR, potentially disrupting the current treatment landscape.

Acoramidis Gains Global Momentum: FDA Approval, EU Recommendation, and Promising Clinical Data

6 months ago

• Acoramidis (Attruby), developed by Stanford Medicine and BridgeBio, receives FDA approval for transthyretin amyloid cardiomyopathy (ATTR-CM) treatment, marking a significant milestone. • The European Medicines Agency's CHMP recommends acoramidis for EU marketing authorization based on positive Phase 3 ATTRibute-CM trial results. • Clinical trials demonstrate acoramidis' efficacy in reducing cardiovascular-related hospitalizations and improving survival rates for ATTR-CM patients. • Bayer and BridgeBio collaborate to commercialize acoramidis, with Bayer holding EU rights and plans for a launch in Europe in early 2025.

BridgeBio's Attruby Receives FDA Approval for ATTR-CM Treatment

6 months ago

• The FDA has approved BridgeBio's Attruby (acoramidis) for treating adults with transthyretin amyloid cardiomyopathy (ATTR-CM), marking a significant milestone for the company. • Attruby is the first and only approved product with a label specifying near-complete stabilization of transthyretin, demonstrating benefits on cardiovascular outcomes. • Clinical trials showed Attruby significantly reduced death and cardiovascular-related hospitalization, while also improving the quality of life for patients with ATTR-CM. • Priced at $225,000 per year, Attruby offers a new treatment option in the ATTR-CM market, though it will compete with existing therapies like Pfizer's tafamidis.

FDA Approves Attruby (Acoramidis) for ATTR-CM, Reducing Cardiovascular Death and Hospitalization

6 months ago

• The FDA has approved Attruby (acoramidis) for treating adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), addressing both wild-type and variant forms of the disease. • Attruby, an oral TTR stabilizer, demonstrated a significant reduction in cardiovascular death and cardiovascular-related hospitalizations in the Phase 3 ATTRibute-CM trial. • The approval marks Attruby as the first and only therapy with a label specifying near-complete stabilization of transthyretin (TTR), targeting the underlying cause of ATTR-CM. • Clinical data showed Attruby improved quality of life and reduced all-cause mortality and cardiovascular hospitalization as early as three months after treatment initiation.

Acoramidis Approved for ATTR-CM, Showing Sustained Benefits in Long-Term Study

6 months ago

• The FDA has approved acoramidis (Attruby) for transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular death and hospitalization. • Acoramidis demonstrated a 42% reduction in all-cause mortality and cardiovascular-related hospitalization events compared to placebo at 30 months. • Long-term data showed early initiation and continuous use of acoramidis over 42 months led to sustained clinical benefits in ATTR-CM patients. • Acoramidis is the first approved product with a label specifying near-complete stabilization of transthyretin (TTR).

FDA Approves UCB's Bimzelx (bimekizumab-bkzx) for Hidradenitis Suppurativa

6 months ago

• The FDA has approved Bimzelx (bimekizumab-bkzx) as the first IL-17A and IL-17F inhibitor for adults with moderate to severe hidradenitis suppurativa (HS). • Approval was based on Phase 3 trials (BE HEARD I and BE HEARD II) demonstrating significant improvement in HS signs and symptoms at Week 16 and sustained responses at Week 48. • Bimekizumab-bkzx showed a higher proportion of patients achieving HiSCR50 (50% improvement) compared to placebo, along with clinically meaningful improvements in HiSCR75. • This approval marks the fifth patient population in the U.S. that may benefit from Bimzelx, addressing a substantial unmet need in HS treatment.

Acoramidis Shows Sustained Benefit in ATTR-CM Patients: Open-Label Extension Data

6 months ago

• Acoramidis demonstrates sustained improvement in cardiovascular outcomes, reducing the risk of cardiovascular hospitalization or all-cause mortality in ATTR-CM patients. • The open-label extension study reveals a statistically significant 36% reduction in all-cause mortality at 36 months with continuous acoramidis treatment. • Early intervention with acoramidis shows early separation from placebo, suggesting a sustained benefit for patients with transthyretin amyloid cardiomyopathy. • Acoramidis continues to be well-tolerated, reinforcing its potential as a first-line treatment option for ATTR-CM, with no new safety concerns identified.

EU Committee Recommends AstraZeneca and Ionis' Wainzua for ATTRv-PN

7 months ago

• The Committee for Medicinal Products for Human Use (CHMP) has recommended Wainzua (eplontersen) for approval in the EU to treat hATTR-PN. • NEURO-TTRansform Phase III trial data showed Wainzua provided sustained benefits in neuropathy impairment and quality of life versus placebo over 66 weeks. • If approved, Wainzua will be the only EU-approved medicine for ATTRv-PN self-administered monthly via auto-injector, offering a novel treatment option. • Eplontersen is also being evaluated in the CARDIO-TTRansform Phase III trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) with over 1,400 participants.

Drug Development Updates