FerroKin BioSciences, Inc. is a clinical stage biotechnology company that specializes in providing services for the treatment of iron-overload in patients with transfusion-dependent hereditary and acquired refractory anemia through the use of iron chelators. The company was established in 2007 by Hugh Young Rienhoff, Jr. and is based in San Carlos, California.

Drugs associated with FerroKin BioSciences, Inc.

Deferitazole magnesium

Target-

Mechanism

Iron chelating agents

Active Org.-

Originator Org.

Shire Development LLC

Active Indication-

Inactive Indication

Beta-Thalassemia [+3]

Drug Highest PhaseDiscontinued

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with FerroKin BioSciences, Inc.

EUCTR2011-005675-16-IT / Not yet recruitingNot Applicable

A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation Therapy - FBS0701 in the treatment of chronic iron overload

Start Date15 May 2012

Sponsor / Collaborator

FerroKin BioSciences, Inc.

EUCTR2011-001718-32-IT / Not yet recruitingNot Applicable

A Phase 2, Open Label, Multi-Center, Single-Dose Pharmacokinetics, and Multiple Dose Study of the Safety, Efficacy and Tolerability of FBS0701 in a Pediatric Population with Transfusional Iron Overload - FBS0701/CTP-07

Start Date07 Sep 2011

Sponsor / Collaborator

FerroKin BioSciences, Inc.

NCT01039636 / CompletedPhase 1

A PHASE 1b OPEN LABEL, MULTI-CENTER, ESCALATING MULTIPLE DOSE STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF FBS0701 IN PATIENTS WITH TRANSFUSIONAL IRON OVERLOAD

The purpose of this research study is to study the safety of increasing doses of FBS0701, and to see how quickly the study medication is absorbed and how quickly it disappears from the bloodstream. FBS0701 is a new, oral iron chelator - a medication taken by mouth that increases the body's elimination of iron. Iron chelators are used in patients who develop iron overload from their transfusions. Four increasing doses of FBS0701 will be tested during this study. The study will start with the lowest dose given to 4 patients (3 mg/kg/day. The next group of 4 patients will receive the next high dose (8mg/kg/day only after the results of the first 4 patients are examined and it is determined safe to continue. Participating patients will take the study medication for 7 days and be followed for 28 days after their last dose to determine if they have any reactions to the study medication - therefore a total of 35 days on study. Patients will need to give up to 17 blood samples over the screening period and first 15 days of the study (a total of about 9 tablespoons). Patients will not need to stay overnight in the clinic but will need to visit the clinic 10 times for screening and on-study visits over the 35 days. Patients currently taking an iron chelator will need to stop that treatment for up to 22 days (up to 5 days before they start the study and for 15 days during the study). The results of this study will be helpful in determining the safety of the drug and the best doses of FBS0701 to be used in the next study which will assess the effectiveness of this new iron chelator.

Start Date01 Nov 2009

Sponsor / Collaborator

FerroKin BioSciences, Inc.

100 Clinical Results associated with FerroKin BioSciences, Inc.

0 Patents (Medical) associated with FerroKin BioSciences, Inc.

Literatures (Medical) associated with FerroKin BioSciences, Inc.

01 Jul 2015·Magnetic Resonance ImagingQ4 · MEDICINE

Liver MRI is more precise than liver biopsy for assessing total body iron balance: a comparison of MRI relaxometry with simulated liver biopsy results

Q4 · MEDICINE

Article

Author: Wood, John C ; Neufeld, Ellis J ; Abi-Saab, Walid ; Rienhoff, Hugh ; Zhang, Pinggao

PURPOSELiver biopsy was long considered the reference standard for measuring liver iron concentration. However, its high sampling variability and invasive nature make it poorly suited for serial analyses. To demonstrate the fallibility of liver biopsy, we use serial estimates of iron chelation efficiency (ICE) calculated by R2 and R2* MRI liver iron concentration (LIC) estimates as well as by simulated liver biopsy (over all physically reasonable sampling variability) to compare the robustness of these three techniques.MATERIALS AND METHODSR2, R2*, transfusional volume, and chelator compliance were obtained from 49 participants in a phase II clinical trial of deferitazole over two years. Liver biopsy LIC results were simulated using sampling errors of 0%, 10%, 20%, 30%, 40% and iron assay variability of 12%. LIC estimates by R2, R2*, and simulated biopsy were used to calculate ICE over time. Bland-Altman limits of agreement were compared across observation intervals of 12, 24, and 48 weeks.RESULTSAt 48 week intervals, LIC estimates by R2, R2* and "perfect" liver biopsy had comparable accuracy in predicting ICE; both MRI methods were superior to any physically realizable liver biopsy (sampling error 10% or higher). LIC by R2* demonstrated the most robust ICE estimates at monitoring intervals of 24 and 12 weeks, but this difference did not remain significant at 48 week intervals.CONCLUSIONMRI relaxometry is superior to liver biopsy for serial LIC observations, such as used in the care of tranfusional siderosis patients, and should also be considered the new standard of LIC determination for regulatory purposes. Among relaxometry techniques, LIC estimates by R2* are more robust for tracking changes in iron balance over intermediate time scales (<=24 weeks).

15 Nov 2013·Blood

Liver MRI Is Better Than Biopsy For Assessing Total Body Iron Balance: Validation By Simulation

Author: Neufeld, Ellis J. ; Wood, John C. ; Rienhoff, Hugh Y. ; Abi-Saab, Walid ; Zhang, Pinggao

AbstractIntroductionLiver iron concentration (LIC) represents the best surrogate of total body iron balance for guiding iron chelation therapy. Liver biopsy was formerly used to determine LIC, but has gradually been replaced by magnetic resonance imaging (MRI) relaxometry because relaxometry is noninvasive and has lower sampling variability. Since neither liver biopsy nor MRI relaxometry can be considered a “gold standard”, there is a need for an independent mechanism to validate their accuracy and reproducibility. In this study, we use serial estimates of iron chelation efficiency calculated by R2 and R2* MRI as well as simulated liver biopsy results to demonstrate that MRI is fundamentally more accurate that liver biopsy in determining iron balance.MethodsAll patients were participating in a phase 2 clinical trial of SPD602 (formerly known as FBS0701) and had strict documentation of iron chelator and blood intake. MRI R2 and R2* LIC estimates were performed at baseline, 12, 24, 48, 72, and 96 weeks. Forty-nine patients completed 24 weeks, 39 completed 48 weeks, and 26 completed 96 weeks of the trial. Liver biopsy LIC results were simulated (by Monte Carlo simulation) using sampling errors having a coefficient of variation (CoV) of 0%, 10%, 20%, 30%, and 40%, and iron assay variability of 12%. LIC estimates by R2, R2*, and simulated biopsy were used to calculate chelation efficiency estimates over time. Bland–Altman limits of agreement were compared across observation timescales of 12, 24, and 48 weeks.ResultsTable 1 summarizes the standard deviation for chelation efficiency estimates performed at 12, 24, and 48 week intervals. For 48 week intervals, R2, R2*, and “perfect” liver biopsy exhibited statistically identical variance (indicated by italic type), and were superior to any “realistic” sampling error for liver biopsy (CoV 10–40%). At shorter measurement intervals, R2* produced the lowest variance estimates. For 12 week intervals, R2* efficiency estimates were confounded by two outliers; exclusion of these two points reduced the standard deviation of 12 week R2* efficiency estimates to 15.6% (p < 0.01).Figure 1 (top row) demonstrates simulated efficiency estimates by “perfect” liver biopsy performed on 12, 24, and 48 week timescales. While “perfect” liver biopsy approaches ideal behavior at 48 week intervals, many non-physiologic estimates (> 100%, < 0%) are produced during shorter term observations. Figure 1 (middle and bottom row) compares the identical relationships for chelation efficiency estimates generated by R2 and R2*, respectively. R2 demonstrates similar scatter as observed for liver biopsy. R2* efficiency estimates are better, with a slope near unity and all points residing in the upper right hand quadrant for 24 and 48 week timescales. Thus both R2 and R2* produce chelation efficiency estimates at least as good as “perfect” liver biopsy and better than biopsy performed with any reasonable estimate of sampling variability.DiscussionEven if liver biopsy is assumed to have no sampling variation, which is unrealistic in clinical practice, MRI relaxometry is superior for tracking of total body iron balance. R2 and R2* are equally robust on annual evaluations, but R2* more closely tracks iron balance on shorter timescales. We conclude that MRI relaxometry should replace liver biopsy for the determination of LIC for both clinical and regulatory purposes.Disclosures:Wood: Shire: Consultancy, Research Funding; Apopharma: Honoraria, Patents & Royalties; Novartis: Honoraria. Zhang:Shire: Employment. Rienhoff:Shire: Consultancy, Milestone Payments Other. Abi-Saab:Shire: Employment, Equity Ownership, Patents & Royalties; AbbVie: Equity Ownership, Patents & Royalties; Novartis: Equity Ownership, Patents & Royalties; Abbott: Equity Ownership, Patents & Royalties; Pfizer: Equity Ownership, Patents & Royalties. Neufeld:Shire: Consultancy.

15 Nov 2013·Blood

R2 and R2* Are Equally Effective In Evaluating Chronic Response To Iron Chelation

Author: Neufeld, Ellis J. ; Wood, John C. ; Rienhoff, Hugh Y. ; Abi-Saab, Walid ; Zhang, Pinggao

AbstractIntroductionMagnetic resonance imaging (MRI) relaxometry, using either R2 or R2* measurements, has generally replaced liver biopsy for estimation of liver iron stores in response to iron chelation therapy, but there have been no longitudinal studies comparing R2 and R2* techniques. We used R2 and R2* liver iron concentration (LICR2 and LICR2*) estimates, transfusional iron burdens, and drug adherence data to calculate iron chelation efficiency in patients undergoing a phase 2 trial of SPD602 (formerly known as FBS0701), a tridentate iron chelator in development. We hypothesized that chelation efficiency estimates derived from LICR2 and LICR2* measurements would be in better agreement than baseline LIC assessments using the two techniques.MethodsIn the phase 2 clinical trial of SPD602, 51 patients underwent a baseline examination, 39 patients completed 48 weeks, and 26 patients completed 96 weeks. MRI assessment of liver R2 and R2* were performed at baseline, 12, 24, 48, 72, and 96 weeks, analyzed by experienced reference laboratories (Ferriscan R2 by Resonance Health Western Australia; R2* by Children's Hospital Los Angeles, CA), and converted to LIC using established calibrations. Efficiency was calculated according to the following equation:where TII is the transfusional iron intake in mg/kg, ΔLIC is the change in LIC in mg/g dry weight, MWdrug and MWFe are the molecular weights of SPD602 and iron respectively, and drug is the total SPD602 consumed in mg/kg.ResultsFigure 1 shows a scattergram of LICR2 versus LICR2* at baseline; both axes were log-transformed to normalize the variance. Resulting linear fit had a slope of 0.996, a scaling factor of 0.114 and an r2 of 0.76 (p< 0.0001). 95% limits of agreement were broad, measuring -53.8% to 53.8%.However, chelation efficiency estimates across the two techniques compared more favorably, with r2 values 0.76 and 0.83 when calculated over 0–48 and 49–96 weeks, respectively. Figure 2 demonstrates the LICR2 and LICR2* chelation efficiency estimates in patients completing 96 weeks. The r2 value was 0.89 with excellent 95% limits of agreement [-3.5 to 3.5%]; elimination of two outliers improved the r2 value to 0.95.DiscussionTaken together, these data illustrate two important points for individual hematology practitioners. First, while R2 and R2* methods are individually as accurate as liver biopsy in predicting true LIC, large discrepancies between LICR2 and LICR2* can be observed for any given individual as shown by the baseline measurements. These discrepancies do not reflect intrinsic deficiencies of either technique, but arise because R2 and R2* are differentially sensitive to pattern and scale of iron deposition in the liver. Thus LICR2 and LICR2* cannot be used interchangeably in an individual.Secondly, however, longitudinal assessments decrease systematic errors between and within techniques, just as paired statistics often provide greater statistical power than unpaired statistics. As a result, both LICR2 and LICR2* produce chelation efficiency estimates that are internally consistent and physiologically reasonable. The choice of MRI assessment technique for clinical trials and for clinical management depends on many logistical considerations, including cost, the expected dynamic range of iron burden, and the availability of trained, experienced personnel for image quality control and analysis. In summary, the present study demonstrates that well-performed LICR2 and LICR2* measurements yield comparable longitudinal monitoring of chelator effectiveness, suitable for clinical trials and for clinical practice.Disclosures:Wood: Shire: Consultancy, Research Funding; Apopharma: Honoraria, Patents & Royalties; Novartis: Honoraria. Zhang:Shire: Employment. Rienhoff:Shire: Consultancy, Milestone Payments Other. Abi-Saab:Shire: Employment, Equity Ownership, Patents & Royalties; AbbVie: Equity Ownership, Patents & Royalties; Novartis: Equity Ownership, Patents & Royalties; Abbott: Equity Ownership, Patents & Royalties; Pfizer: Equity Ownership, Patents & Royalties. Neufeld:Shire: Consultancy.

News (Medical) associated with FerroKin BioSciences, Inc.

21 Nov 2022

·www.fiercebiotech.com

Merck & Co. inks $1.4B blood cancer biotech buyout to muscle in on Bristol Myers, Novartis

The Merck takeover marks a second exit for a team led by Hugh Rienhoff, Jr., M.D. Merck & Co. has opened its wallet wide to buy a midphase treatment for life-threatening diseases of the bone marrow, paying $1.35 billion to snap up Imago BioSciences for its oral lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat. Imago discovered the small molecule and took it into phase 2 on the strength of evidence that inhibiting LSD1 reduces the hallmark symptoms of myeloproliferative neoplasms (MPNs), a family of bone marrow cancers that can transform into acute myeloid leukemia. If Imago is right, bomedemstat will improve outcomes in the MPNs essential thrombocythemia, myelofibrosis and polycythemia vera. Merck has bought into the idea. To take control of Imago, the Big Pharma has agreed to pay $36 per share in cash in a deal that values its quarry at $1.35 billion. Imago’s stock closed at $17.40 on Friday and has ranged from $11 to $27 since the biotech went public last year at $16 per share. Imago raised $340 million across its private rounds and IPO. The deal, which Merck expects to close in the first quarter of 2023, opens up several markets for the Big Pharma. Imago is running phase 2 clinical trials in essential thrombocythemia and myelofibrosis, and, with enrollment now complete, plans to share data updates from both studies next month. The biotech has already met with the FDA to discuss an active-controlled phase 3 trial in essential thrombocythemia. Imago sees opportunities to improve on the best-available therapy in its lead indications, noting that the current options in the large essential thrombocythemia market are poorly tolerated and that there is no disease modifying therapy for myelofibrosis. The biotech sees polycythemia vera, which it is studying in a third phase 2 trial, as a growing market in need of new well-tolerated treatments. To make a big dent on its target markets, bomedemstat will need to demonstrate an edge over Jakafi, which is sold by Incyte in the U.S. and Novartis overseas, and Bristol Myers Squibb’s JAK inhibitor Inrebic. Merck could also face competition from other LSD1 inhibitors such as Iadademstat, although Oryzon, the developer of that molecule, has focused its clinical activities on acute myeloid leukemia. The takeover marks a second exit for a team led by Hugh Rienhoff Jr., M.D. The Imago CEO is one of several people in the biotech’s C-suite who previously held leadership positions at FerroKin BioSciences, a rare disease player that Shire bought for $100 million upfront a decade ago.

AcquisitionPhase 2Phase 3IPO

100 Deals associated with FerroKin BioSciences, Inc.

100 Translational Medicine associated with FerroKin BioSciences, Inc.

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Deferitazole magnesium	Iron Overload More	Pending

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