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Castle Biosciences Presents Second Study Confirming Ability of DecisionDx-Melanoma to Inform Sentinel Lymph Node Biopsy Decisions at the 8th International Congress on Cancer Metastasis

On October 28, 2019 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported the presentation of data supporting clinical use of the DecisionDx-Melanoma test to inform decisions of whether to discuss or recommend the sentinel lymph node biopsy (SLNB), a surgical procedure used to provide additional prognostic information (Press release, Castle Biosciences, OCT 28, 2019, View Source [SID1234549927]). The study found that DecisionDx-Melanoma test results can be used with clinicopathologic factors to identify patients at low risk of sentinel lymph node (SLN) positivity. This information can inform patient discussions and recommendations regarding the SLNB surgical procedure in line with national melanoma clinical practice guidelines.

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The study titled, "Identification of T1-T2 melanoma patients at low risk for a positive sentinel lymph node using the 31-gene expression profile test," was presented during an Oral Abstract session at the 8th International Congress on Cancer Metastasis (ICCM) in San Francisco, October 25-27.

Study Background:

National guidelines recommend the SLNB surgical procedure to assess prognosis of melanoma patients whose tumor features suggest at least a 5% likelihood of sentinel lymph node (SLN) positivity. The guidelines do not recommend the procedure if a patient has a likelihood of SLN positivity of less than 5%.
Among all patients with T1-T2 melanoma (tumor depth of 2 mm or less), only 5-10% have a positive SLNB, with the likelihood of a positive SLN decreasing with age.
The DecisionDx-Melanoma test is a 31-gene expression profile prognostic test for cutaneous melanoma that predicts 5-year risk of metastasis as low risk (Class 1, 1A lowest risk) or high risk (Class 2, 2B highest risk), as well as metastasis to the sentinel lymph node.
The DecisionDx-Melanoma test has been previously validated to identify patients with T1-T2 melanoma with SLN positivity rates below 5%, thus helping guide SLNB discussions and recommendations.
This study was designed to further evaluate the ability of the DecisionDx-Melanoma test to identify T1-T2 melanoma patients with low risk for a positive SLN, using the combination of the previously published cohort with a novel cohort, totaling 1,905 prospectively tested consecutive T1-T2 melanoma patients. The cohort had a median age of 64 years, median Breslow depth of 1.2 millimeters and 13% had ulceration present.
Key Findings:

In patients with T1-T2 tumors of any age (DecisionDx-Melanoma intended use population) and a Class 1A test result, SLN positivity was 4.9%, significantly less than patients with a Class 1B-2A result (p<0.01) or a Class 2B result (p<0.0001) and below the 5% threshold at which guidelines do not recommend the procedure.
In SLNB-assessed patients with T1-T2 tumors 65 years of age or older and a Class 1A test result, SLN positivity was 2.7%, significantly less than patients with a Class 1B-2A (p<0.01) or Class 2B result (p<0.0001), and below the 5% threshold at which guidelines do not recommend the procedure.
In SLNB-assessed patients with T1 tumors (tumor depth of 1 mm or less) 65 years of age or older and a Class 1A test result, SLN positivity was 1.0%, significantly less than patients with a Class 1B-2A (p<0.01) or a Class 2B result (p<0.001), and below the 5% threshold at which guidelines do not recommend the procedure.
Use of the DecisionDx-Melanoma test to guide SLNB decisions in patients with T1-T2 melanoma could reduce SLNB surgical procedures by 58% in patients 65 years of age or older, as 367 of 629 patients who underwent this procedure had a Class 1A result.
In a multi-center, retrospective dataset of 576 T1-T2 patients with long-term follow-up (median 7.5 years) Class 1A patients with T1-T2 melanoma had melanoma-specific survival of 99.7%, overall survival of 97.3%%, distant metastasis-free survival of 94.9% and recurrence-free survival of 92.9% at five years, supporting that this population can safely avoid the SLNB surgical procedure.
In a multi-center, prospective dataset of 246 T1-T2 patients with long-term follow-up (median 3.2 years), Class 1A patients with T1-T2 melanoma had overall survival of 99.4%, distant metastasis-free survival of 98.7% and recurrence-free survival of 96.6% at three years, adding further support that this population can safely avoid the SLNB surgical procedure.
"The data presented at the ICCM show the DecisionDx-Melanoma test, in combination with patient age and clinical features, can identify T1-T2 melanoma patients who are at low risk for a positive sentinel lymph node and can avoid the SNLB surgical procedure, based on thresholds established by national clinical melanoma guidelines," said Federico A. Monzon, M.D., FCAP, chief medical officer at Castle Biosciences. "Additionally, T1-T2 melanoma patients with a DecisionDx-Melanoma Class 1A result show very low rates of metastatic recurrence and melanoma specific death."

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 3,900 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and five prospective risk of recurrence studies including more than 780 patients. Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter study cohorts that included more than 2,000 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results.

Blueprint Medicines Announces FDA Intent to Split Avapritinib New Drug Application into Separate Submissions for PDGFRA Exon 18 Mutant GIST and Fourth-Line GIST

On October 28, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported an update regarding its New Drug Application (NDA) for avapritinib for the treatment of adults with PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and fourth-line GIST (Press release, Blueprint Medicines, OCT 28, 2019, View Source [SID1234549926]).

On October 25, 2019, Blueprint Medicines received written feedback from the U.S. Food and Drug Administration (FDA) following its NDA mid-cycle review meeting. The FDA informed Blueprint Medicines that it intends to administratively split the proposed indications for avapritinib into two separate NDAs, one for PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and one for fourth-line GIST. Given the acceleration of the ongoing Phase 3 VOYAGER clinical trial of avapritinib in patients with third- and fourth-line GIST and the anticipated availability of top-line data in the second quarter of 2020, the FDA requested top-line data from the VOYAGER trial. The FDA indicated these data would be informative in its review of the proposed fourth-line indication and potential clinical benefit in this population, including its evaluation of response rate and safety for the fourth-line indication. An extension of the review period for the fourth-line GIST NDA will likely be required to enable Blueprint Medicines to provide the top-line VOYAGER data to the FDA.

"Throughout the development of avapritinib, we have had a productive and collaborative dialogue with the FDA about the potential of avapritinib to address important medical needs in subsets of patients with advanced GIST," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "We plan to continue to work closely with the FDA during its review of the separate NDAs for PDGFRA Exon 18 mutant GIST and fourth-line GIST, and we plan to submit the requested VOYAGER trial data as expeditiously as possible."

Blueprint Medicines has completed patient screening in the VOYAGER trial of avapritinib in patients with third- and fourth-line GIST and expects to complete patient enrollment by the end of November 2019 and report top-line data in the second quarter of 2020. Blueprint Medicines plans to prioritize completion of the VOYAGER trial and delay initiation of its COMPASS-2L trial in second-line GIST. Subject to an initial approval of avapritinib, Blueprint Medicines plans to submit a supplemental NDA to the FDA for avapritinib for third-line GIST in the second half of 2020.

"We are focusing our strategy and resourcing for the avapritinib clinical development program on three core activities: completing the VOYAGER trial and supporting the FDA’s review of NDAs for PDGFRA Exon 18 mutant GIST and fourth-line GIST, submitting an NDA for advanced systemic mastocytosis in the first quarter of 2020, and submitting an NDA for third-line GIST in the second half of 2020," said Andy Boral, MD, PhD, Chief Medical Officer of Blueprint Medicines. "We remain committed to exploring the potential of avapritinib in patients with second-line GIST and working with the GIST community to advance precision medicine approaches that can transform patient care, both of which may be further enabled by a robust dataset from the VOYAGER trial including nearly 500 patients with third- and fourth-line GIST."

About Avapritinib

Avapritinib is an investigational, oral precision therapy that selectively and potently inhibits KIT and PDGFRA mutant kinases. It is a type 1 inhibitor designed to target the active kinase conformation; all oncogenic kinases signal via this conformation. Avapritinib has demonstrated broad inhibition of KIT and PDGFRA mutations associated with GIST, including potent activity against activation loop mutations that are associated with resistance to currently approved therapies.

Blueprint Medicines is initially developing avapritinib for the treatment of advanced GIST, advanced systemic mastocytosis (SM), and indolent and smoldering SM. The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation and one for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.

About GIST

GIST is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal (GI) tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.

Most GIST cases are caused by a spectrum of clinically relevant mutations that force the KIT or PDGFRA protein kinases into an increasingly active state. Because currently available therapies primarily bind to the inactive protein conformations, certain primary and secondary mutations typically lead to treatment resistance and disease progression.

In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Mutational testing is critical to tailor therapy to the underlying disease driver and is recommended in expert guidelines. Currently, there are no approved therapies for patients with KIT-driven GIST whose disease progresses beyond imatinib, sunitinib and regorafenib. In patients with metastatic PDGFRA D842V-driven GIST, progression occurs in a median of approximately three to four months with available therapy.

About the Phase 3 VOYAGER Clinical Trial

The VOYAGER clinical trial is a global, open-label, randomized, Phase 3 trial designed to evaluate the safety and efficacy of avapritinib versus regorafenib in patients with third- or fourth-line advanced GIST. Eligible patients will have previously received imatinib and one or two additional tyrosine kinase inhibitors. The trial is designed to enroll approximately 460 patients randomized 1:1 to receive avapritinib dosed at 300 mg once daily (QD) or regorafenib dosed at 160 mg QD for three weeks, followed by one week off, at multiple sites in the United States, European Union, Australia and Asia. Patients who are randomized to receive regorafenib and experience disease progression confirmed by central radiology review may be offered the opportunity to cross-over to the avapritinib treatment arm. The primary efficacy endpoint is progression free survival determined by central radiologic assessment per modified Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. Secondary endpoints include objective response rate, overall survival and quality of life outcome measures. Regorafenib, also known as Stivarga, is an oral, multi-kinase inhibitor approved by the FDA for the treatment of patients with third-line GIST. Additional trial details are also available on www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT03465722).

Akebia Therapeutics to Report Third Quarter 2019 Financial Results and Discuss Recent Business Highlights

On October 28, 2019 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for people with kidney disease, reported plans to release its financial results for the third quarter ended September 30, 2019, on Tuesday, November 12, 2019 before the opening of the financial markets (Press release, Akebia, OCT 28, 2019, View Source [SID1234549925]).

Akebia will host a conference call at 9:00 a.m. Eastern Time on Tuesday, November 12th, to discuss its third quarter financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 9996464. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through November 18, 2019. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 9996464. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2019 Financial Results

On October 28, 2019 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the third quarter ended September 30, 2019 (Press release, CRISPR Therapeutics, OCT 28, 2019, View Source [SID1234549924]).

"In 2019, we’ve made significant progress across several development programs, including ongoing enrollment of our CTX001 trials in beta thalassemia and severe sickle cell disease, with preliminary data expected from these programs later this year. We also began treating patients in our clinical trial for CTX110, our allogeneic CAR-T therapy, and are advancing additional CAR-T candidates toward clinical development," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Looking forward, we expect a robust 2020, with continued focus on execution as we anticipate conducting five clinical trials in parallel."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

Enrollment is ongoing at six clinical trial sites in the U.S., Canada and Europe for the Phase 1/2 study of CTX001 in patients with transfusion-dependent beta thalassemia (TDT) and at ten clinical trial sites in the U.S., Canada and Europe for the study in patients with severe sickle cell disease (SCD).

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion for orphan drug designation (ODD) of CTX001 for the treatment of TDT. In addition, we are expanding the TDT patient population for CTX001 to include beta zero/beta zero subtypes.

The Company expects to release preliminary safety and efficacy data from the ongoing Phase 1/2 clinical trials in late 2019.

Immuno-Oncology

The Company has begun treating patients in a Phase 1/2 trial to assess the safety and efficacy of CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies. The multi-center, open label trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110. The study is currently enrolling at five clinical trial sites in the U.S. and Australia. In addition, the Company obtained approval from Health Canada for its Clinical Trial Application (CTA). The Company believes its CRISPR-based allogeneic CAR-Ts may have a superior product profile compared to current autologous therapies and allow accessibility to broader patient populations.

The Company expects to initiate a Phase 1/2 clinical trial of CTX120, targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma, in the first half of 2020. CRISPR Therapeutics continues to advance additional allogeneic CAR-T candidates toward clinical development including CTX130, targeting CD70 for the treatment of solid tumors and hematologic malignancies. The Company continues to scale its capabilities to enable rapid advancement of these programs into and through the clinic.

The Company recently announced it entered into a license agreement with KSQ Therapeutics whereby CRISPR Therapeutics gained access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ gained access to CRISPR Therapeutics’ IP for editing novel gene targets identified by KSQ as part of its current and future eTILTM (engineered tumor infiltrating lymphocyte) cell programs. The agreement further strengthens the Company’s proprietary allogeneic CAR-T platform.

The Company will present a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference on November 9, 2019 related to single-cell RNA sequencing and functional assessment of healthy donor and cancer patient-derived T and CAR-T cells (#P187).

Regenerative Medicine

On September 17, 2019, CRISPR Therapeutics, in collaboration with ViaCyte, presented positive in vitro data for a potential immune-evasive cell replacement therapy for diabetes at the 55th Annual Meeting of the European Association for the Study of Diabetes (EASD) in Barcelona, Spain. The oral presentation included new data that demonstrate the successful differentiation of CRISPR-edited human pluripotent stem cells to pancreatic precursor cells.

Other Corporate Matters

Vertex has exercised the options granted under the collaboration it established with CRISPR Therapeutics in 2015 to in-license three additional targets for the development of treatments using CRISPR-based gene editing. The targets include the cystic fibrosis transmembrane conductance regulator (CFTR) gene and two undisclosed targets. Under the terms of the agreement, CRISPR Therapeutics will receive an upfront payment of $30 million in connection with the option exercise and has the potential to receive up to $410 million in development, regulatory and commercial milestones and royalties on net product sales for each of the three targets, and Vertex will receive exclusive rights to develop and commercialize products related to these targets globally. The research term of the Company’s 2015 collaboration with Vertex has now expired, and Vertex no longer holds rights to in-license additional targets under that agreement.

The Company recently announced proposed plans that Casebia Therapeutics, previously a joint venture between CRISPR Therapeutics and Bayer, would operate under the direct management of CRISPR Therapeutics. Upon closing of the transaction, Casebia Therapeutics would focus on the development of its lead programs in hemophilia, ophthalmology and autoimmune diseases, with Bayer having opt-in rights for two products at IND submission. The transaction is subject to negotiation and execution of definitive agreements as well as certain customary conditions. The Company and Bayer are negotiating the definitive agreements and, subject to the finalization of the definitive agreements and satisfaction of closing conditions, anticipate to close the transaction in the fourth quarter of 2019.
Third Quarter 2019 Financial Results

Cash Position: Cash as of September 30, 2019, was $629.7 million, compared to $427.9 million as of June 30, 2019, an increase of $201.8 million as increased cash operating expenses were offset by $68.6 million net proceeds from financing activities and $175 million upfront payments received from Vertex related to the 2019 collaboration agreement announced in June.

Revenues: Total collaboration revenues were $211.9 million for the third quarter of 2019 compared to $0.6 million for third quarter of 2018 with the increase primarily driven by the collaboration agreement with Vertex.

R&D Expenses: R&D expenses were $57.2 million for the third quarter of 2019 compared to $39.8 million for the third quarter of 2018. The increase was driven by increased headcount and services expense supporting the advancement of the hemoglobinopathies program, the broadening of the Company’s wholly-owned immuno-oncology portfolio, as well as increased investment in the Company’s CRISPR/Cas9 platform research and some non-cash expense related to the Company’s collaboration with Vertex.

G&A Expenses: General and administrative expenses were $15.5 million for the third quarter of 2019 compared to $10.2 million for the third quarter of 2018. The increase was driven by increased headcount-related expense and external professional and consulting service expense.

Net Income (Loss): Net Income was $138.4 million for the third quarter of 2019 compared to a loss of $50.7 million for the third quarter of 2018, driven predominantly by increased revenue recognized in connection with the Company’s collaboration with Vertex.

Actinium Announces Positive Interim Results from Iomab-B Pivotal Phase 3 SIERRA Trial at 50% of Total Patient Enrollment

On October 28, 2019 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported key interim findings from the pivotal Phase 3 SIERRA trial of Iomab-B, including feasibility and safety data, at 50% of total patient enrollment (Press release, Actinium Pharmaceuticals, OCT 28, 2019, View Source [SID1234549923]). The SIERRA trial (Study of Iomab-B in Elderly Relapse/Refractory Acute Myeloid Leukemia) is a 150-patient, 1:1 randomization Phase 3 pivotal trial that is studying Iomab-B (Iodine-131 apamistamab) compared to physician’s choice of salvage chemotherapy in patients age 55 and above with active, relapsed or refractory AML (Acute Myeloid Leukemia). These interim data provide information on safety and feasibility of using Iomab-B to enable a BMT (Bone Marrow Transplant), the only curative treatment option in this patient population that is not typically considered eligible for BMT.

Key findings from the first 75 patients enrolled (50% of total enrollment) in the trial include:

– Patients enrolled in the SIERRA trial were a median age of 64 (55-76) and had active disease with a median bone marrow blast percentage at randomization of 28 percent (5-97%). All patients were intermediate or poor, adverse risk groups. Two-thirds (68%) of the patients enrolled were in the poor prognostic, adverse cytogenetic and molecular risk group.

– All patients receiving a therapeutic dose of Iomab-B (31/31) in the study arm received a bone marrow transplant and engrafted rapidly without delay in blood count recovery.

– Only 7/38 patients (18%) randomized to the control arm achieved an initial complete response (CR) and were able to proceed to a conventional bone marrow transplant.

– Of the 31 patients who failed the primary endpoint in the control arm (82% failure), 20 (64%) were considered eligible for potential rescue by treatment with Iomab-B followed by a BMT. All 20 patients (100%) who crossed over and received a therapeutic dose of Iomab-B followed by a BMT achieved engraftment without delay in blood count recovery despite high blast count burden prior to transplant (median 35%).

– 12/38 patients (32%) randomized to the control arm received targeted agents all of which were recently approved. 11/12 patients (92%) received the Bcl-2 inhibitor venetoclax with either an hypomethylating agent or low dose cytarabine. Of these, only 3/11 patients (27%) achieved a CR.

– 100-day Non-Relapse Transplant Related Mortality remains lower in the Iomab-B arm (1/31 patients or 3%) compared to patients in the control arm who received conventional transplants (2/7 or 29%). Of the 31 patients who received Iomab-B in the study arm, 30 are potentially evaluable for the primary endpoint compared to 5 in the control arm. This difference between study arm and control arm remains consistent with that reported at the interim update at 25% of enrollment.

– The SIERRA trial remains the only randomized Phase 3 clinical trial to offer BMT as an option for patients age 55 and above with active, relapsed or refractory AML.

Sergio Giralt, M.D., Chief of Adult BMT, Memorial Sloan Kettering Cancer Center; Chair, Myeloma Service, said, "The results from the first 50% of patients to be enrolled in the SIERRA trial continue to be highly encouraging, particularly Iomab-B’s ability to enable a transplant in this patient population that would otherwise be ineligible. Despite eight new therapies having been approved for patients with AML, better outcomes for patients with AML are needed, in particular for the large number of relapsed and refractory patients. Newly approved targeted agents are not curative and as seen in the SIERRA trial do not enable a high rate of potentially curative BMT."

*Footnotes for table can be found below Conference Call and Webcast information

Mark Berger, M.D., Actinium’s Chief Medical Officer, said, "We are thrilled that data from the halfway point of the SIERRA trial validate the promising interim safety and feasibility results from the first 25% of patients in this trial. It is particularly gratifying for me to see that these relapsed, refractory patients with heavy disease burden who received a therapeutic dose of Iomab-B were successfully transplanted. In addition, it is heartening that Iomab-B can be an effective pathway to transplant even when the recently approved targeted therapies unfortunately fail, as shown by our control arm and crossover data. With the first half of the trial behind us and with strong data in hand, we are focused on taking this message to hematologists and transplant physicians. We look forward to continuing to execute on our strategies to bring Iomab-B to patients as quickly as possible."

Conference Call and Webcast Information

Time and Date: 12:00 PM ET on Monday, October 28th
Registration Link: https://platform.cinchcast.com/ses/kSO55A9SLNGFoAvv1hgHpg~~
U.S./Canada Toll Free Dial-in: (855) 698-6739
Participant Dial-in: (646) 402-9440
Conference ID: 0417

A replay of the call will be available on the Investor Relations page of the Company’s website.

Table Footnotes:

Data available on 31 patients per arm
1 patient with circulating blasts and bone marrow <5% not included in median/range
No therapeutic dose (6) due to: declining KPS (3), infusion reaction (1), unfavorable biodistribution (1), post-randomization eligibility (1)
Ineligible for crossover (9) due to: hospice care/progression (4), declined/ineligible for BMT (2), died pre-crossover (3). Eligible for crossover (2), received dosimetry but not Iomab-B therapy due to declining status
Crossed-over and received therapeutic dose of Iomab-B
ANC engraftment data not available (1), platelet engraftment data not available (5)
ANC and platelet engraftment data not available (1), engraftment failure (1)
ANC engraftment data not available (2), platelet engraftment data not available (4)
1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab-B and BMT when stable
Data available on: Iomab-B study arm (30), CR after salvage therapy (7) and no CR after salvage crossed-over received Iomab-B and transplanted (16)
About the SIERRA Trial

The SIERRA trial (Study of Iomab-B in Elderly Relapse/Refractory Acute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. Iomab-B is an ARC (Antibody Radiation-Conjugate) comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume.