All Seven Sufferers Handled with BEAM-101 Achieved Hemoglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Discount to
Preliminary Security Profile In keeping with Busulfan Conditioning and Autologous Hematopoietic Stem Cell Transplantation
All Seven Sufferers Dosed Achieved Goal (NYSE:) Cell Dose with One or Two Mobilization Cycles and Skilled Fast Neutrophil and Platelet Engraftment
Markers of Hemolysis Normalized or Improved in All Sufferers
Beam to Host Investor Occasion on Dec. 8, 2024, at 8 p.m. PT
SAN DIEGO, Dec. 07, 2024 (GLOBE NEWSWIRE) — Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology firm creating precision genetic medicines by means of base modifying, at this time introduced new security and efficacy information from its BEACON Section 1/2 scientific trial of BEAM-101 in sufferers with sickle cell illness (SCD) with extreme vaso-occlusive crises (VOCs). The information had been featured at this time within the press program for the 66th American Society of Hematology (ASH) Annual Assembly and Exposition in San Diego and shall be introduced in an oral session on Sunday, Dec. 8, 2024, at 10 a.m. PT.
In keeping with Beam’s beforehand introduced information, up to date information from seven sufferers handled with investigational base-editing remedy BEAM-101 demonstrated sturdy and sturdy will increase in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), speedy neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. No VOCs had been reported post-engraftment. A abstract of the outcomes from the continuing scientific research is supplied beneath.
These preliminary information from the BEACON trial are very encouraging and spotlight the potential of BEAM-101 to ship significant scientific advantages to sufferers with extreme sickle cell illness, stated Matthew M. Heeney, M.D., affiliate chief of hematology at Dana-Farber/Boston Kids’s Most cancers and Blood Issues Middle. The information from the primary seven sufferers exhibit the power for BEAM-101 to dramatically modify the hemoglobin profile to precise a majority of protecting fetal hemoglobin. All sufferers mobilized effectively and had speedy engraftment with a low variety of neutropenic days. I sit up for the continued maturation of the information to supply additional insights into the long-term advantages of BEAM-101 for individuals residing with sickle cell illness.
It is an honor to share the preliminary outcomes from BEACON with the hematology neighborhood on the ASH Annual Assembly, the place there may be broad recognition of the numerous burden that sickle cell illness locations on sufferers and their households, stated John Evans, chief government officer of Beam. We consider these early information for BEAM-101 are a testomony to the potential of our base-editing expertise to supply a differentiated possibility for sickle cell sufferers, having demonstrated a strong improve in fetal hemoglobin of >60%, a lower in hemoglobin S to
To this point, greater than 35 sufferers have cleared screening and enrolled within the BEACON Section 1/2 scientific trial, and of those, 11 sufferers have been dosed with BEAM-101. As of an Oct. 28, 2024, information cut-off, a complete of seven sufferers with extreme SCD had been handled with BEAM-101 and included within the security and efficacy evaluation with comply with up starting from 1 to 11 months.
Key highlights embrace the next:
- Fast and Sustained Will increase in Protecting Fetal Hemoglobin (HbF): All sufferers achieved endogenous HbF ranges exceeding 60% and discount in corresponding sickle hemoglobin (HbS) beneath 40% that was sturdy. A pancellular distribution of HbF was noticed after the elimination of transfused blood.
- Strong and Sustained Whole (EPA:) Hemoglobin (Hb) Ranges: Whole hemoglobin ranges elevated quickly with decision of anemia in sufferers after elimination of the transfused blood.
- Environment friendly Cell Assortment and Fast Engraftment: All sufferers achieved the minimal goal cell dose in both 1 or 2 cycles of mobilization (common: 1.4). The imply time to neutrophil engraftment was 17.1 days (vary: 15“21), with a low imply period of neutropenia (6.3 days). The imply time to platelet engraftment was 19.1 days (vary: 11“34).
- Normalization of Hemolysis Markers: Key markers of hemolysis, together with oblique bilirubin, haptoglobin, lactate dehydrogenase and reticulocytes, normalized or improved in all sufferers following BEAM-101 therapy.
- Security Profile In keeping with Busulfan and Autologous Hematopoietic Stem Cell Transplantation (HSCT): The security profile of BEAM-101 was in step with busulfan conditioning and autologous HSCT. The most typical treatment-emergent adversarial occasions (TEAEs) had been in step with busulfan conditioning, together with febrile neutropenia, stomatitis and anemia. One affected person died 4 months after BEAM-101 infusion because of respiratory failure that was decided by the investigator to be possible associated to busulfan conditioning and deemed unrelated to BEAM-101. No VOCs had been reported post-engraftment.
ASH Investor Occasion Data
Beam will host a dwell and webcast investor occasion on Dec. 8, 2024, at 8:00 p.m. PT in San Diego to overview the important thing displays from this 12 months’s ASH assembly. The occasion shall be webcast dwell and could be accessed beneath Occasions & Shows within the Traders part of the corporate’s web site at www.beamtx.com. The archived webcast shall be obtainable on the corporate’s web site starting roughly two hours after the occasion.
About BEAM-101
BEAM-101 is an investigational genetically modified cell remedy for the therapy of extreme sickle cell illness (SCD). The one-time remedy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) which were base-edited within the promotor areas of the HBG1/2 genes and are administered through a hematopoietic stem cell transplant process. The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter with out disrupting BCL11A expression, resulting in elevated manufacturing of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the results of naturally occurring variants seen in hereditary persistence of fetal hemoglobin. HbF is the predominant hemoglobin variant throughout improvement and formative years. The security and efficacy of BEAM-101 is being evaluated within the ongoing BEACON Section 1/2 research, an open-label, single-arm, multicenter trial in grownup sufferers with SCD with extreme vaso-occlusive crises (VOCs).
About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology firm dedicated to establishing the main, totally built-in platform for precision genetic medicines. To attain this imaginative and prescient, Beam has assembled a platform with built-in gene modifying, supply and inner manufacturing capabilities. Beam’s suite of gene modifying applied sciences is anchored by base modifying, a proprietary expertise that’s designed to allow exact, predictable and environment friendly single base modifications, at focused genomic sequences, with out making double-stranded breaks within the DNA. This has the potential to allow a variety of potential therapeutic modifying methods that Beam is utilizing to advance a diversified portfolio of base modifying applications. Beam is a values-driven group dedicated to its individuals, cutting-edge science, and a imaginative and prescient of offering life-long cures to sufferers affected by critical illnesses.
Cautionary Be aware Relating to Ahead-Trying Statements
This press launch comprises forward-looking statements throughout the which means of the Non-public Securities Litigation Reform Act of 1995. Traders are cautioned to not place undue reliance on these forward-looking statements, together with, however not restricted to, statements associated to: the therapeutic functions and potential of our expertise, together with with respect to SCD; our plans, and anticipated timing, to advance our applications; the scientific trial designs and expectations for BEAM-101 and ESCAPE; our displays on the ASH annual assembly; and our means to develop life-long, healing, precision genetic medicines for sufferers by means of base modifying. Every forward-looking assertion is topic to vital dangers and uncertainties that would trigger precise outcomes to vary materially from these expressed or implied in such assertion, together with, with out limitation, dangers and uncertainties associated to: our means to develop, get hold of regulatory approval for, and commercialize our product candidates, which can take longer or value greater than deliberate; our means to lift extra funding, which is probably not obtainable; our means to acquire, preserve and implement patent and different mental property safety for our product candidates; the uncertainty that our product candidates will obtain regulatory approval essential to provoke human scientific trials; that preclinical testing of our product candidates and preliminary or interim information from preclinical research and scientific trials is probably not predictive of the outcomes or success of ongoing or later scientific trials; that initiation and enrollment of, and anticipated timing to advance, our scientific trials might take longer than anticipated; that our product candidates or the supply modalities we depend on to manage them might trigger critical adversarial occasions; that our product candidates might expertise manufacturing or provide interruptions or failures; dangers associated to aggressive merchandise; and the opposite dangers and uncertainties recognized beneath the headings Threat Components Abstract and Threat Components in our Annual Report on Kind 10-Okay for the 12 months ended December 31, 2023, our Quarterly Reviews on Kind 10-Q for the quarterly interval ended September 30, 2024 and in any subsequent filings with the Securities and Change Fee. These forward-looking statements communicate solely as of the date of this press launch. Components or occasions that would trigger our precise outcomes to vary might emerge every so often, and it isn’t potential for us to foretell all of them. We undertake no obligation to replace any forward-looking assertion, whether or not because of new data, future developments or in any other case, besides as could also be required by relevant regulation.
Contacts:
Traders:
Holly Manning
Beam Therapeutics
hmanning@beamtx.com
Media:
Dan Budwick
1AB
dan@1abmedia.com
