Dafinchi AI

BioMarin Pharmaceutical Inc.

BMRN

Healthcare

2w

Progress and Expectations for BMN 333 Long-Acting CNP Therapy for Achondroplasia

Encouraging pharmacokinetic results showing free CNP levels more than 3x greater than previous long-acting CNP agents in healthy volunteers.
Plan to initiate dose-finding Phase II/III study in the first half of 2026 with a targeted approval in 2030.
Preclinical data and genetic evidence suggest potential for greater growth with increased CNP exposure.
Study design aims for a meaningful differentiation in growth outcomes, with a focus on health and wellness markers beyond height.

Neurocrine Biosciences, Inc.

NBIX

Healthcare

2w

Rapid and Early Adoption of CRENESSITY in CAH Patients

CRENESSITY launched in late December 2024 and has already generated $53 million in Q2 2025 sales, more than tripling Q1 sales.
Over 1,000 treatment forms received since launch, with 75% reimbursement rate.
Prescriptions written by diverse providers including multidisciplinary centers, pediatric and adult endocrinologists.
Most prescribers have initiated treatment for only 1-2 patients, indicating early-stage adoption.
Strong presence at ENDO conference with positive clinician feedback and data presentations.

Neurocrine Biosciences, Inc.

NBIX

Healthcare

2w

Financial Performance Summary

CRENESSITY sales grew from $15 million in Q1 2025 to $53 million in Q2 2025, reflecting strong early adoption.
Gross to net was impacted by contracting and co-pay assistance programs, particularly for INGREZZA.
INGREZZA delivered $624 million in Q2 sales, with 15% sequential growth and 8% year-over-year growth.
INGREZZA saw record new patient starts and total prescriptions, with prescription market share gains in the first half of 2025.
Neurocrine Biosciences reported $682 million in net product sales during Q2 2025, representing 17% year-over-year growth.
Over 75% of CRENESSITY dispensed prescriptions were reimbursed in Q2 2025.
There was a $5 million inventory build in the quarter reflecting channel growth for CRENESSITY.

Mirum Pharmaceuticals, Inc.

MIRM

Healthcare

1w

Pipeline Progress and Key Study Readouts in 2025-2026

Mirum's pipeline is advancing rapidly, with three pivotal studies expected to read out over the next 24 months, including the VISTAS Phase IIb study in PSC, with topline data expected in Q2 2026.
The VISTAS study is on track to complete enrollment this quarter, with interim analysis last year providing confidence in IBAT inhibition's potential in PSC.
Additional studies, VANTAGE in PBC and EXPAND in cholestatic pruritus, are progressing well, with enrollment expected to complete in 2026.
The company is also initiating a Phase II study of MRM-3379 for Fragile X syndrome, with FDA feedback received and enrollment planned to start by year-end.

Apellis Pharmaceuticals, Inc.

APLS

Healthcare

2w

FDA Label Expansion for EMPAVELI in C3G and Primary IC-MPGN

FDA approved a label expansion for EMPAVELI for patients 12 years and older with C3 myelopathy and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
This is the company's third FDA approval in 4 years, highlighting scientific strength and regulatory success.
The approval enables treatment with a C3-targeting therapy that achieves outcomes across proteinuria reduction, eGFR stabilization, and C3 deposit clearance.
Launch is underway, expanding into rare nephrology and underserved populations, including pediatric patients and post-transplant recurrence.

Schrödinger, Inc.

SDGR

Healthcare

1w

Progress and Strategic Opportunities for SGR-1505 MALT1 Inhibitor

Initial Phase I data for SGR-1505 showed a well-tolerated profile with responses in heavily pretreated CLL and Waldenstrom's macroglobulinemia patients, with 3 of 17 CLL patients responding and all 5 Waldenstrom's patients responding.
The program has received FDA Fast Track designation, highlighting its potential as a promising therapeutic in refractory hematologic cancers.
Schrodinger is exploring strategic opportunities to accelerate clinical development and maximize the potential of SGR-1505, rather than pursuing independent late-stage trials.
The company plans to provide further updates on dose escalation, translational data, and regulatory feedback later this year.
The emerging profile of SGR-1505 suggests best-in-class potential, with early evidence supporting its use in refractory disease, especially in patients previously exposed to BTK and BCL2 inhibitors.

Castle Biosciences, Inc.

CSTL

Healthcare

2w

FDA Breakthrough Designation for DecisionDx-Melanoma and Strategic Path to FDA Approval

Castle Biosciences received FDA Breakthrough Device Designation for DecisionDx-Melanoma, signaling a significant regulatory milestone.
The company is actively progressing towards an FDA submission, with no specific public timeline disclosed.
Management expressed confidence that existing data supports FDA approval, emphasizing the importance of the breakthrough designation in accelerating regulatory pathway.

Kura Oncology, Inc.

KURA

Healthcare

1w

FDA Priority Review and Regulatory Progress for Ziftomenib

The FDA has accepted Kura's NDA for ziftomenib in relapsed/refractory NPM1-mutant AML, granting a priority review with a PDUFA date of November 30, 2025.
Management reports ongoing collaborative and constructive interactions with the FDA, emphasizing alignment with the review timeline.
Regulatory milestones include addressing information requests and preparing for pre-approval inspections, with a focus on ensuring a successful review outcome.

Seres Therapeutics, Inc.

MCRB

Healthcare

1w

Advancement and Clinical Development of SER-155

Seres has advanced preparation for the next stage of SER-155 development, including a Phase II protocol submission to the FDA.
The Phase Ib study showed a 77% relative risk reduction in bloodstream infections in allo-HSCT patients, supporting further development.
The upcoming Phase II study is designed to enroll approximately 248 patients with an adaptive design and interim analysis, with results expected within 12 months of initiation.
Positive interactions with the FDA and breakthrough therapy designation facilitate the study design and potential approval pathway.
The company aims to use interim data to inform next steps, including potential engagement with the FDA for a registrational study.

Inovio Pharmaceuticals, Inc.

INO

Healthcare

5d

Regulatory Progress and BLA Submission Strategy for INO-3107

Inovio remains on track to submit its BLA for INO-3107 in the second half of 2025, with a goal of file acceptance by year-end.
The company has completed the design verification testing of the CELLECTRA 5PSP device, a key regulatory milestone.
Inovio has requested a rolling submission of its BLA under breakthrough therapy designation, aiming for a 6-month review period and a potential PDUFA date around mid-2026.
The company successfully completed an FDA inspection of its clinical trial operations, an important step in regulatory compliance.
Differences in trial design and technology, such as DNA medicine versus viral vectors, distinguish INO-3107 from competitors' programs.

Bionano Genomics, Inc.

BNGO

Healthcare

3d

Potential Strategic Partnerships and Acquisition Discussions Ongoing

Management acknowledged ongoing discussions with potential strategic partners and the interest from the market.
Bionano highlighted its advanced position in the space, with a strong financial profile and strategic focus.
The company emphasized its commitment to its digital pathology transformation strategy.
While not explicitly seeking acquisitions, Bionano remains open to strategic collaborations that align with its goals.
The company’s visibility and market presence make it a target for strategic interest, but no specific deals were disclosed.