A Multicenter, Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Brentuximab Vedotin plus Doxorubicin, Vinblastine and Dacarbazine in Indian Patients with Untreated Stage 3/4 Classical Hodgkin Lymphoma - NIL

Start Date30 Jul 2024

Sponsor / Collaborator

Baxalta US, Inc.

NCT04985682

/ CompletedPhase 4

Phase 4, Multicenter, Prospective, Interventional, Post-Marketing Study in Hemophilia A Patients in India Receiving ADVATE as On-Demand or Prophylaxis Under Standard Clinical Practice

The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.

Start Date14 Jan 2022

Sponsor / Collaborator

Baxalta, Inc.

[+1]

CTRI/2021/10/037406

/ Active, not recruitingPhase 4

Phase 4, Multicenter, Prospective, Interventional, Post-Marketing Study in Hemophilia A Patients in India Receiving ADVATE as On-Demand or Prophylaxis Under Standard Clinical Practice - Nil

Start Date01 Nov 2021

Sponsor / Collaborator

Baxalta US, Inc.

[+1]

100 Clinical Results associated with Baxalta, Inc.

0 Patents (Medical) associated with Baxalta, Inc.

Literatures (Medical) associated with Baxalta, Inc.

01 Aug 2021·Journal of clinical immunologyQ2 · MEDICINE

Steady-State Serum IgG Trough Levels Are Adequate for Pharmacokinetic Assessment in Patients with Immunodeficiencies Receiving Subcutaneous Immune Globulin

Q2 · MEDICINE

Article

Author: Engl, Werner ; Li, Zhaoyang ; McCoy, Barbara ; Yel, Leman

Abstract:

Patients with primary immunodeficiency diseases often require lifelong immunoglobulin (IG) therapy. Most clinical trials investigating IG therapies characterize serum immunoglobulin G (IgG) pharmacokinetic (PK) profiles by serially assessing serum IgG levels. This retrospective analysis evaluated whether steady-state serum IgG trough level measurement alone is adequate for PK assessment. Based on individual patient serum IgG trough levels from two pivotal trials (phase 2/3 European [NCT01412385] and North American [NCT01218438]) of weekly 20% subcutaneous IG (SCIG; Cuvitru, Ig20Gly), trough level-predicted IgG AUC (AUCτ,tp) were calculated and compared with the reported AUC calculated from serum IgG concentration-time profiles (AUCτ). In both studies, mean AUCτ,tpvalues for Ig20Gly were essentially equivalent to AUCτwith point estimates of geometric mean ratio (GMR) of AUCτ,tp/AUCτnear 1.0 and 90% CIs within 0.80–1.25. In contrast, for IVIG, 10%, mean AUCτ,tpvalues were lower than AUCτby >20%, (GMR [90% CI]: 0.74 [0.70–0.78] and 0.77 [0.73–0.81] for the two studies, respectively). Mean AUCτ,tpvalues calculated for 4 other SCIG products (based on mean IgG trough levels reported in the literature/labels) were also essentially equivalent to the reported AUCτ(differences <10% for all except HyQvia, a facilitated SCIG product), while differences for IVIG products were >20%. In conclusion, steady-state serum IgG levels following weekly SCIG remain stable, allowing for reliable prediction of AUC over the dosing interval using trough IgG levels. These findings indicate that measuring steady-state serum IgG trough levels alone may be adequate for PK assessment of weekly SCIG.

01 Apr 2021·BloodQ1 · MEDICINE

Rurioctocog alfa pegol PK-guided prophylaxis in hemophilia A: results from the phase 3 PROPEL study

Q1 · MEDICINE

Article

Author: Engl, Werner ; Klamroth, Robert ; Savage, William ; Windyga, Jerzy ; Stasyshyn, Oleksandra ; Collins, Peter W. ; Radulescu, Vlad ; Ewenstein, Bruce ; Tangada, Srilatha D. ; Ibrahim, Hishamshah Mohd

Abstract:

Rurioctocog alfa pegol prophylaxis targeting factor VIII (FVIII) troughs ≥1% has shown to be efficacious with an acceptable safety profile in people with hemophilia A (PwHA). The PROPEL trial compared safety and efficacy of 2 target FVIII troughs in PwHA aged 12 to 65 years, with severe disease, annualized bleeding rate ≥2, and previous FVIII treatment. PwHA were randomized to 12 months’ pharmacokinetic (PK)-guided rurioctocog alfa pegol prophylaxis targeting FVIII troughs of 1% to 3% (reference arm) or 8% to 12% (elevated arm); first 6 months was treatment-adjustment period. The primary endpoint was absence of bleeds during the second 6 months, analyzed using multiple imputations (full analysis set [FAS]). In the 1% to 3% and 8% to 12% arms, respectively, point estimates (95% confidence interval) of proportions of PwHA with zero total bleeds were 42% (29% to 55%) and 62% (49% to 75%) in FAS (N = 115; P = .055) and 40% (27% to 55%) and 67% (52% to 81%) in per-protocol analysis set (N = 95; P = .015). Dosing frequency and consumption varied in each arm. Adverse events (AEs) occurred in 70/115 (60.9%) PwHA; serious AEs in 7/115 (6%) PwHA, including 1 treatment-related in 8% to 12% arm (transient anti–FVIII inhibitor). There were no deaths, serious thrombotic events, or AE-related discontinuations. PK-guided prophylaxis was achievable and efficacious in both arms. No new safety signals were observed in the 8% to 12% arm. These results demonstrate elevated FVIII troughs can increase the proportion of PwHA with zero bleeds and emphasize the importance of personalized treatment. This trial was registered at www.clinicaltrials.gov as #NCT02585960.

11 Feb 2021·BloodQ1 · MEDICINE

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Q1 · MEDICINE

Article

Author: Samulski, R. Jude ; Walsh, Christopher E. ; McPhee, Scott W. J. ; Young, Guy ; Bilic, Ivan ; Chapin, John C. ; Scheiflinger, Friedrich ; Josephson, Neil C. ; Ewenstein, Bruce ; Monahan, Paul E. ; von Drygalski, Annette ; Reipert, Birgit M. ; de la Rosa, Maurus ; Konkle, Barbara A. ; Rottensteiner, Hanspeter ; Escobar, Miguel A.

Abstract:

Gene therapy has the potential to maintain therapeutic blood clotting factor IX (FIX) levels in patients with hemophilia B by delivering a functional human F9 gene into liver cells. This phase 1/2, open-label dose-escalation study investigated BAX 335 (AskBio009, AAV8.sc-TTR-FIXR338Lopt), an adeno-associated virus serotype 8 (AAV8)–based FIX Padua gene therapy, in patients with hemophilia B. This report focuses on 12-month interim analyses of safety, pharmacokinetic variables, effects on FIX activity, and immune responses for dosed participants. Eight adult male participants (aged 20-69 years; range FIX activity, 0.5% to 2.0%) received 1 of 3 BAX 335 IV doses: 2.0 × 1011; 1.0 × 1012; or 3.0 × 1012 vector genomes/kg. Three (37.5%) participants had 4 serious adverse events, all considered unrelated to BAX 335. No serious adverse event led to death. No clinical thrombosis, inhibitors, or other FIX Padua–directed immunity was reported. FIX expression was measurable in 7 of 8 participants; peak FIX activity displayed dose dependence (32.0% to 58.5% in cohort 3). One participant achieved sustained therapeutic FIX activity of ∼20%, without bleeding or replacement therapy, for 4 years; in others, FIX activity was not sustained beyond 5 to 11 weeks. In contrast to some previous studies, corticosteroid treatment did not stabilize FIX activity loss. We hypothesize that the loss of transgene expression could have been caused by stimulation of innate immune responses, including CpG oligodeoxynucleotides introduced into the BAX 335 coding sequence by codon optimization. This trial was registered at www.clinicaltrials.gov as #NCT01687608.

126

News (Medical) associated with Baxalta, Inc.

07 May 2026

·www.prnewswire.com

BioLife Solutions Reports First Quarter 2026 Financial Results

Total revenue of $27.5 million, up 25% over Q1 2025 GAAP gross margin and non-GAAP adjusted gross margin of 64% GAAP net income of $1.2 million and non-GAAP adjusted EBITDA of $6.2 million or 22% of revenue Conference call begins at 4:30 p.m. Eastern time today BOTHELL, Wash., May 7, 2026 /PRNewswire/ -- BioLife Solutions, Inc. (Nasdaq: BLFS ) ("BioLife" or the "Company"), a leading developer and supplier of cell processing tools and services for the cell and gene therapy ("CGT") market, announces financial results for the three months ended March 31, 2026. "We delivered a solid start to 2026, with first quarter revenue up 25% year-over-year and adjusted EBITDA of $6.2 million," said Roderick de Greef, Chairman and Chief Executive Officer of BioLife Solutions. "Healthy demand for our biopreservation media and growth across our broader product portfolio reinforces our market leading position, while high-margin, recurring revenue drives a strong financial profile. Our products are embedded across a significant portion of commercial therapies and clinical pipelines, and we are seeing continued momentum across the CGT landscape including expansion into larger indications, encouraging data readouts, renewed funding activity and strategic M&A. This positions us well for the next phase of growth in this market and we remain confident in our full year outlook." First Quarter 2026 Business Highlights Our biopreservation media is utilized in approximately 250 ongoing commercially sponsored clinical trials in the U.S., representing a more than 70% market share. This includes over 30 Phase III trials, or nearly 80% of these late-stage trials. Our CellSeal vials and hPL products are used in over 35 clinical trials. Our biopreservation media is embedded in 17 unique commercial CGTs as of March 31, 2026, with expectations that approvals for 9 additional products, geographic expansions, earlier lines of treatment, or new indications will occur over the next 12 months. Our CellSeal cryogenic vials and hPL products are embedded in four approved therapies. First Quarter 2026 Financial Results BioLife is presenting various financial metrics under U.S. generally accepted accounting principles (GAAP) and as adjusted (non-GAAP). In addition, BioLife completed the divestiture of evo in 2025, and is presenting its financial condition and operating results as discontinued operations for all periods presented within the Unaudited Condensed Consolidated Balance Sheets and Unaudited Condensed Consolidated Statements of Operations. The Unaudited Condensed Consolidated Statements of Comprehensive Income (Loss) and Unaudited Condensed Consolidated Statements of Cash Flows are presented on a consolidated basis for both continuing operations and discontinued operations. All amounts, percentages, and disclosures for all periods presented reflect only the continuing operations of the Company unless otherwise noted. REVENUE Total revenue for the first quarter of 2026 was $27.5 million, an increase of $5.4 million, or 25%, from $22.1 million for the first quarter of 2025 and up $2.7 million, or 11%, from the fourth quarter of 2025. GROSS MARGIN Gross margin (GAAP) for the first quarter of 2026 was 64% compared with 67% for the first quarter of 2025. Adjusted gross margin (non-GAAP) for the first quarter of 2026 was 64% compared with 68% for the first quarter of 2025. OPERATING INCOME / (LOSS) Operating income (GAAP) for the first quarter of 2026 was $27 thousand compared with an operating loss of $0.5 million for the first quarter of 2025. Adjusted operating income (non-GAAP) for the first quarter of 2026 was $1.0 million compared with $1.2 million for the first quarter of 2025. NET INCOME Net income (GAAP) for the first quarter of 2026 was $1.2 million compared with $0.3 million for the first quarter of 2025. Adjusted net income (non-GAAP) for the first quarter of 2026 was $2.1 million compared with $2.0 million for the first quarter of 2025. NET INCOME PER SHARE Net income per share (GAAP) for the first quarter of 2026 was $0.02 compared with $0.01 for the first quarter of 2025. ADJUSTED EBITDA Adjusted EBITDA, a non-GAAP measure, for the first quarter of 2026 was $6.2 million, or 22% of revenue, compared with $5.4 million, or 24% of revenue, for the first quarter of 2025. CASH, CASH EQUIVALENTS, AND MARKETABLE SECURITIES Cash, cash equivalents, and marketable securities as of March 31, 2026, were $111.5 million. 2026 Financial Guidance BioLife is reiterating its 2026 financial guidance as follows: Revenue of $112.5 million to $115.0 million, representing growth of 17% to 20% compared with 2025 revenue from continuing operations; Gross margin (GAAP) and adjusted gross margin (non-GAAP) in the mid-60% range; Net income (GAAP) for the full year; and Continued expansion of adjusted EBITDA margin (non-GAAP). Conference Call & Webcast Management will discuss the Company's financial results, provide a business update and answer questions during a conference call and live webcast today at 4:30 p.m. ET (1:30 p.m. PT). To access the webcast, log onto the Investor Relations page of the BioLife Solutions website at . In addition, the conference call will be accessible by dialing toll-free 1-833-630-0431 or 1-412-317-1808 for international callers. A webcast replay will be available approximately two hours after the call ends and will be archived on for 90 days. About BioLife Solutions BioLife is a leading developer and supplier of cell processing tools and services for the CGT market. Our expertise facilitates the commercialization of new therapies by supplying solutions that maintain the health and function of biologic materials during the collection, development, manufacturing and distribution. For more information, please visit , and follow BioLife on LinkedIn and X. Cautions Regarding Forward Looking Statements Certain statements contained in this press release are not historical facts and may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "plans," "expects," "believes," "anticipates," "designed," "may," "estimate," "guidance," and similar words are intended to identify forward-looking statements. Forward-looking statements are based on our current expectations and beliefs, and involve a number of risks and uncertainties that are difficult to predict and that could cause actual results to differ materially from those stated or implied by the forward-looking statements. A description of certain of these risks, uncertainties and other matters can be found in filings we make with the U.S. Securities and Exchange Commission, all of which are available at . Because forward-looking statements involve risks and uncertainties, actual results and events may differ materially from results and events currently expected by us. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. We undertake no obligation to publicly update these forward-looking statements to reflect events or circumstances that occur after the date hereof or to reflect any change in its expectations with regard to these forward-looking statements or the occurrence of unanticipated events. Non-GAAP Measures of Financial Performance To supplement our financial statements, which are presented on the basis of U.S. generally accepted accounting principles (GAAP), the following non-GAAP measures of financial performance are included on a consolidated basis in this release: adjusted gross margin, adjusted operating expenses, adjusted operating income/(loss), adjusted net income/(loss), earnings before interest, taxes, depreciation and amortization (EBITDA), and adjusted EBITDA. A reconciliation of GAAP to adjusted non-GAAP financial measures is included as an attachment to this press release. We believe these non-GAAP financial measures are useful to investors in assessing our operating performance. We use these financial measures internally to evaluate our operating performance and for planning and forecasting of future periods. We also believe it is in the best interests of investors to provide this non-GAAP information. While we believe these non-GAAP financial measures provide useful supplemental information to investors, there are limitations associated with the use of these non-GAAP financial measures. These non-GAAP financial measures may not be reported by competitors, and they may not be directly comparable to similarly titled measures of other companies due to differences in calculation methodologies. The non-GAAP financial measures are not an alternative to GAAP information and are not meant to be considered in isolation or as a substitute for comparable GAAP financial measures. They should be used only as a supplement to GAAP information and should be considered only in conjunction with our consolidated financial statements prepared in accordance with GAAP. Media & Investor Relations At the Company Troy Wichterman Chief Financial Officer (425) 402-1400 [email protected] Investors Alliance Advisors IR Jody Cain (310) 691-7100 [email protected] SOURCE BioLife Solutions, Inc. 21% more press release views with Request a Demo

Financial Statement

05 May 2026

·www.businesswire.com

4D Path Announces Strategic Financing and Appointment of Katherine Vega Stultz and Robert J. Hombach to Its Board of Directors

NEWTON, Mass.--(BUSINESS WIRE)--4D Path, a company dedicated to personalizing cancer care through a novel, physics-informed approach to predict tumor response to therapy, today announced the first close of a strategic financing round to accelerate the development of treatment predictive biomarkers for 4D Path-sponsored clinical use and partnerships with Biopharma companies. 4D Path’s strategy is to develop digital treatment prediction biomarkers that inform more clear and accurate treatment selection earlier in the disease process. 4D Path has pan-cancer potential and is six for six in delivering clinically relevant results across breast, ovarian, colorectal, and skin cancers, across stages (e.g., early-stage and metastatic), with existing standards of care (chemotherapy, immunotherapy) and investigational therapies. Katherine Vega Stultz and Robert “Bob” J. Hombach have been appointed to the Board of Directors to strengthen and diversify the organization as it enters a new stage of exciting, accelerated growth. Ms. Stultz brings several decades of leadership in strategy, clinical development and commercialization, building high-performing teams and delivering therapies from bench to bedside across modalities and diseases including oncology. She has previously served in Board capacities in private and public companies. Ms. Stultz will serve as 4D Path’s Board Chair. Mr. Hombach is a highly accomplished financial and operational executive with three decades of leadership in the healthcare sector. His experience includes serving on the Audit, Finance and Compensation Committees for several public and private Boards of Directors, such as FORTUNE 500 companies like BioMarin (NASDAQ: BMRN), as well as Seaport Therapeutics. “We are excited to add Katherine and Bob to our board. Katherine’s deep expertise in oncology strategy, R&D, and commercialization coupled with Bob’s company strategy, financial and operational expertise perfectly complement the outstanding group of leaders on our board,” said Jacopo Leonardi, CEO of 4D Path. “Their addition coupled with our strategic financing will serve as a catalyst to bring this promising treatment predictive technology one step closer to helping patients around the world beat cancer.” Ms. Stultz has launched several multi-billion dollar products and directed the clinical project organization worldwide at Celgene overseeing over 30 mid/late stage clinical programs across hematology and oncology. Ms. Stultz is currently CEO and President of BrainXell Therapeutics Global Inc. “I am thrilled to be a part of the 4D Path growth story as the company embarks on further generating clinical evidence and advancing partnerships to better identify the most appropriate treatment approach for an individual patient. This is a critical unmet need for medical oncologists and a development challenge as companies progress their clinical strategies.” Mr. Hombach served as the Executive Vice President, Chief Financial Officer and Chief Operations Officer of Baxalta (NYSE: BXLT), a $6 billion global biopharmaceutical company, until it was acquired by Shire plc. in 2016. He was instrumental in Baxalta’s successful spin off from its parent company, Baxter (NYSE: BAX), a nearly $18 billion company, where he previously served as Corporate Vice President and Chief Financial Officer. "I am very excited to join the Board of 4D Path as the company builds a compelling growth trajectory of tremendous potential benefit for patients, providers, drug developers, and investors." About 4D Path 4D Path has created a groundbreaking platform, the patented Q-Plasia OncoReader (QPOR™), designed to directly measure and quantify cell cycle deregulation and tumor immune microenvironment dynamics to predict a patient’s response to therapy. This unprecedented view into tumor dynamics creates new pathways from clinical use to research and development applications—bringing more effective, personalized therapies to patients. 4D Path is focused on a bold vision where every person impacted by cancer is diagnosed quickly and accurately and receives the best, most personalized treatment plan to beat cancer and live a longer, healthier life.

21 Apr 2026

·www.prnewswire.com

BioLife Solutions Wins INTERPHEX 2026 Biotech Innovation Award for CellSeal® CryoCase

Award Recognizes Technologies That Address Important Industry Needs and Advance Prior Standards BOTHELL, Wash., April 21, 2026 /PRNewswire/ -- BioLife Solutions, Inc. (NASDAQ: BLFS), a leading developer and supplier of bioproduction products and services for the cell and gene therapy ("CGT") market, today announced it has received the INTERPHEX 2026 Biotech Innovation Award for its CellSeal® CryoCase, a rigid cryogenic containment system purpose-built for frozen biologic materials. Continue Reading Frozen CellSeal® CryoCase, BioLife Solutions’ rigid cryogenic containment system designed to protect biologic materials during freezing, storage, and distribution. CellSeal CryoCase was developed in response to documented container failures in CGT manufacturing, where flexible cryobags have historically been susceptible to stress fractures and seal fatigue from cycles of controlled rate freezing, liquid nitrogen storage, and distribution. CryoCase provides reinforced structural protection across the full frozen product journey while remaining compatible with closed system manufacturing workflows. As CGT programs scale toward commercialization, regulators are paying closer attention to how frozen products are handled and inspected. CryoCase supports visual inspection for container integrity and potential particulates while giving manufacturers greater confidence in containment across product development. "We're pleased to receive this recognition from INTERPHEX, one of the industry's leading forums for pharmaceutical and biotechnology innovation," said Roderick de Greef, CEO of BioLife Solutions. "This award is meaningful validation that CryoCase addresses a real need in the market. We specifically built it to address containment challenges the industry is currently facing." Launched in 2025, CryoCase is available as part of BioLife's CellSeal® portfolio. For more information, visit . About BioLife Solutions BioLife Solutions is a leading developer and supplier of bioproduction products and services for the cell and gene therapy (CGT) and broader biopharma markets. Our expertise facilitates the commercialization of new therapies by supplying solutions that maintain the health and function of biologic materials during collection, development, storage, and distribution. For more information, please visit or follow BioLife on LinkedIn and X. Media & Investor Relations At the Company Troy Wichterman Chief Financial Officer (425) 402-1400 [email protected] Investors Alliance Advisors IR Jody Cain (310) 691-7100 [email protected] SOURCE BioLife Solutions, Inc. 21% more press release views with Request a Demo

Gene Therapy

100 Deals associated with Baxalta, Inc.

100 Translational Medicine associated with Baxalta, Inc.

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Pipeline

Pipeline Snapshot as of 12 May 2026

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Phase 2

Phase 3

Approved

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

Coagulation Factor IX (Recombinant) ( factor IX )	Hemophilia B More	Approved
Recombinant Human Coagulation Factor VIII (Takeda) ( F10 )	Hemorrhage More	Phase 3
Vonicog Alfa (Baxalta) ( vWF )	Hemorrhage More	Phase 3
SHP-654 ( F8 )	Hemophilia A More	Phase 1/2
BAX-335 ( factor IX )	Hemophilia B More	Phase 1/2

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