—New Drug Application submission planned for second quarter of 2022—

—Full data set to be presented at an upcoming medical meeting—

 

SAN MATEO, Calif.–(BUSINESS WIRE)– Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company dedicated to delivering targeted therapies for rare cancers, today announced positive topline data from the pivotal Phase 3 MOMENTUM study—a global, randomized, double-blind clinical trial evaluating momelotinib (MMB) in myelofibrosis patients who are symptomatic and anemic and previously treated with an approved JAK inhibitor. The trial met all of its primary and key secondary endpoints.

“These data are extremely exciting and everything we had hoped to see from the trial,” said Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology. “To achieve statistically significant and clinically important efficacy across all prespecified primary and key secondary endpoints while maintaining platelet counts in such a difficult to treat patient population is remarkable, and a confirmation of the anemia response we identified in the comprehensive review of our previous Phase 3 studies.”

Topline data announced based on 195 patients (MMB n = 130; DAN n = 65) include:

• Primary Endpoint of Total Symptom Score (TSS) of >50%: 25% in the MMB arm vs. 9% in the control arm (p=0.0095)
• Secondary Endpoint of Transfusion Independence (TI): 31% in the MMB arm vs. 20% in the control arm (one-sided p=0.0064; non-inferiority)
• Secondary Endpoint of Splenic Response Rate (SRR) >35%: 23% in the MMB arm vs. 3% in the control arm (p=0.0006)
• The rate of Grade 3 or worse adverse events in the randomized treatment period was 54% in the MMB arm and 65% in the control arm. Serious treatment emergent adverse events were 35% in the MMB arm and 40% in the control arm.
• Mean baseline characteristics for all patients were TSS of 27, Hemoglobin (Hgb) of 8 g/dL and platelet count of 145 x 10 ⁹/L
• The full data set will be presented at an upcoming medical meeting

“As a clinician, I am thrilled to see data that confirm the potential of momelotinib as a treatment option for myelofibrosis patients who are anemic or at risk of becoming anemic,” said Ruben Mesa, MD, FACP, Executive Director of the Mays Cancer Center, home to UT Health San Antonio, MD Anderson Cancer Center, and co-Principal Investigator of the study. “Anemia of myelofibrosis is strongly correlated with reduced quality of life and a decrease in overall survival. Half of all myelofibrosis patients present with anemia at diagnosis and virtually all become anemic over time. With currently approved therapies being myelosuppressive, it’s wonderful to know that we may soon have such an effective treatment option for these patients.”

Barbara Klencke, MD, Chief Medical Officer of Sierra Oncology, stated, “We are committed to working tirelessly to bring momelotinib to patients as quickly as possible. We would like to thank the patients and investigators who participated in this study and look forward to presenting the full data set at an upcoming medical meeting.”

Conference Call & Webcast
In connection with this announcement, Sierra will host a conference call and webcast on Tuesday, January 25, 2022, at 8:00 am ET. The call may be accessed by calling (833) 927-1758 (Toll-free in North America) or +1 (929) 526-1599 (International Dial-in) and entering the Conference ID number: 007608. The call will be webcast live and will be accessible through the Investor section of the Company’s website at www.SierraOncology.com. An archived replay of the webcast will be made available at the same location.

About Momelotinib
Momelotinib is a potent, selective and orally bioavailable JAK1, JAK2 and ACVR1 / ALK2 inhibitor under investigation for the treatment of myelofibrosis in symptomatic, anemic patients previously treated with an approved JAK inhibitor. More than 1,200 subjects have received momelotinib since clinical studies began in 2009, including approximately 1,000 patients treated for myelofibrosis, several of whom remain on treatment for over 11 years. Momelotinib is the first and only JAK inhibitor to demonstrate positive data for all key hallmarks of the disease—symptoms, splenic response and anemia.

About Myelofibrosis
Myelofibrosis is a rare blood cancer that results from dysregulated JAK-STAT signaling and is characterized by constitutional symptoms, splenomegaly (enlarged spleen) and progressive anemia. From prior studies with momelotinib, we know approximately half of myelofibrosis patients are moderately to severely anemic when eligible for JAK inhibitor treatment. Furthermore, currently approved JAK inhibitors only address symptoms and splenomegaly and are myelosuppressive. This can lead to worsening anemia, resulting in dose reductions that potentially reduce treatment effect.

About the Pivotal MOMENTUM Clinical Trial
MOMENTUM is a global, randomized, double-blind Phase 3 clinical trial of momelotinib versus danazol in patients with myelofibrosis who were symptomatic and anemic, and had been previously treated with an FDA-approved JAK inhibitor. The study was designed to evaluate the safety and efficacy of momelotinib for the treatment and reduction of the key hallmarks of disease: symptoms, blood transfusions (due to anemia) and splenomegaly (enlarged spleen).

The primary endpoint of the study is Total Symptom Score (TSS) reduction of >50% over the 28 days immediately prior to the end of Week 24 compared to baseline TSS, using the Myelofibrosis Symptom Assessment Form (MFSAF). Secondary endpoints included Transfusion Independence (TI) rate for >12 weeks immediately prior to the end of Week 24 with Hgb levels ≥ 8 g/dL, and Splenic Response Rate (SRR) based on splenic volume reduction of >35% at Week 24. The study enrolled 195 patients based on a planned 180 patients across 21 countries.

Danazol was selected as the treatment comparator given its use to ameliorate anemia in patients with myelofibrosis, as recommended by National Comprehensive Cancer Network (NCCN) and European Society of Medical Oncology (ESMO) guidelines. Patients were randomized 2:1 (MMB n = 130 and DAN n = 65) to receive either momelotinib or danazol. After 24 weeks of treatment, patients on danazol were allowed to crossover to receive momelotinib. Early cross-over to momelotinib was available for confirmed symptomatic splenic progression.

About Sierra Oncology
Sierra Oncology is a late-stage biopharmaceutical company on a mission to deliver targeted therapies that treat rare forms of cancer. We harness our deep scientific expertise to identify compounds that target the root cause of disease to advance targeted therapies with assets on the leading edge of cancer biology. Our team takes an evidence-based approach to understand the limitations of current treatments and explore new ways to change the cancer treatment paradigm. Together we are transforming promise into patient impact.

For more information, visit www.SierraOncology.com.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Sierra Oncology’s expectations regarding the regulatory timeline, presentation of the results of the MOMENTUM trial, commercialization and future success of momelotinib, the company’s potential opportunity in myelofibrosis, the company’s ability to identify compounds and statements by the company’s Chief Medical Officer and the Executive Director of the Mays Cancer Center. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements. Such forward-looking statements are subject to risks and uncertainties, including, among others, the factors described under the heading “Risk Factors” set forth in Sierra Oncology’s filings with the Securities and Exchange Commission from time to time. Sierra Oncology undertakes no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220125005390/en/

PHARMAESSENTIA’S BESREMi® (ROPEGINTERFERON ALFA-2B-NJFT) NOW AVAILABLE FOR THE TREATMENT OF PEOPLE WITH POLYCYTHEMIA VERA IN THE UNITED STATES

BESREMi® is the only therapy indicated for adults with PV regardless of treatment history

PharmaEssentia introduces holistic services program to support prescribed patients

December 6, 2021, Burlington, MA – PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that BESREMi® (ropeginterferon alfa-2b-njft) is now commercially available in the U.S. to eligible patients with polycythemia vera (PV). BESREMi was approved by the FDA in November as the only interferon for adults with polycythemia vera. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

“Today marks the beginning of a new chapter in the treatment of PV. Our team is delivering on our goal to bring an innovative solution that may help more people manage not only the symptoms of PV, but target the disease itself to gain durable control with potential to reduce progression over time,” said Meredith Manning, U.S. General Manager. “We look forward to working closely with U.S. providers to raise awareness of this therapy and help advance treatment goals.”

PharmaEssentia SOURCE Now Available to Support People with PV in the U.S.

With the commercial availability of BESREMi, PharmaEssentia is also launching a comprehensive patient support program, which can be found at www.pharmaessentiaSOURCE.com.

Learn more

U.S. FDA APPROVES BESREMi® (ROPEGINTERFERON ALFA-2B-NJFT) AS THE ONLY INTERFERON FOR ADULTS WITH POLYCYTHEMIA VERA

With deep, durable control demonstrated by over 7.5 years of clinical data, BESREMi® can be used at any point in the PV journey to support treatment goals

Milestone represents PharmaEssentia’s first approved indication in the United States

November 12, 2021, Burlington, MA – PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that the U.S. Food and Drug Administration (FDA) has approved BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with polycythemia vera (PV).

BESREMi is an innovative monopegylated, long-acting interferon, which exhibits its cellular effects in polycythemia vera in the bone marrow. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic, and infections disorders. PharmaEssentia is preparing to make BESREMi available in the coming weeks in the U.S.

Learn more

In 1992, I was a Technical Service Representative for a major chemical company, working with clinical laboratories, when I learned through my yearly medical checkup that my platelet count was significantly elevated.  A CBC confirmed what I suspected, essential thrombocythemia (ET). “Enjoying” the first of many bone marrow biopsies proved the diagnosis to be correct.  I met with a pathologist friend and he explained the prognosis.  Except for the elevated platelets I had no other symptoms.

Fast forward to 2004.  I’m having bloodwork checked by a hematologist prior to minor surgery.  I still had no physical symptoms of a myeloproliferative disease.  He came back into the waiting room and announced, “You have morphed into myelofibrosis.”   I didn’t want to believe it, but I knew as a certified medical technologist, MT(ASCP), that the cells don’t lie!  Years of nervous waiting followed, until in 2009 I began to experience terrible pruritis and increasing fatigue.  It was time to move to the next step.

I checked with Mayo Clinic in Scottsdale, Dr. Reuben Mesa, and with M.D. Anderson in Houston, Dr. Srdan Verstovsek.  Sure enough, the myelofibrosis was progressing and I was positive for the JAK-2 mutation.  “Dr. V” told me that a new Phase III study was to start, and that an oncologist/hematologist in San Antonio was enrolling in the study.  That was good news.  San Antonio was much closer to home!

I found my hero, Dr. Roger Lyons, now retired.   After more bloodwork, more bone marrow biopsies, he thought I was a good candidate for the Comfort I study, the first Phase III ruxolitinib study.  It was of course a doubleblind study, but by the third week of the study I knew that I had not drawn a placebo!  The itching began to cease and I felt normal. I stayed in the study through completion, and continued on what came to be called Jakafi for quite a while after.  It was a miracle!  The first drug successfully developed to treat myelofibrosis!

Then there was a problem.  In 2018, I began to develop growths on my nose.  No one suspected it had anything to do with Jakafi. When every dermatologist had exhausted the list of probable diagnoses, Dr. Lyons gave permission to discontinue the Jakafi to see what would happen.  Again, as when I started ruxolitinib, I knew very quickly that Jakafi was the culprit.  Not good news, but the positive effects of the Jakafi stayed with me for over two years.  Luck is really on my side.

Pruritis began anew in 2020, my white blood count had begun to increase, and Dr. Lyons had retired, but fortune smiled again.  Dr. Mesa, who I’d seen years ago at Mayo Clinic Scottsdale, had come to the University of Texas Health Science Center San Antonio as the head of a new partnership with M.D. Anderson Cancer Center.  As my new doctor he suggested we try Jakafi again and adjust the dose as required.  Now I take 20 mg Jakafi daily.  I’m feeling well, considering my 82 years.

Chicago, IL, November 1, 2021 (Newswire.com) – In realworld usage and in clinical trials, interferon (IFN) in a variety of forms has shown high rates of hematologic and molecular responses in many patients with rare blood cancers known as myeloproliferative neoplasms (MPNs). Other patients have no significant response, and some develop drug resistance to recombinant interferon over time.

A report summarizing a three-year Interferon Initiative, released today by the MPN Research Foundation, sheds light on the reasons for these varying responses, and may have impactful applications not only for MPNs − including polycythemia vera, essential thrombocythemia and myelofibrosis − but also for other blood cancers and solid tumors.

“The initiative brought together multi-institutional investigations by key researchers from across the US, Europe and Australia to uncover a deeper understanding of the mechanisms by which interferon works for patients with an MPN,” said Barbara Van Husen, MPNRF board chair.

Read more

A literature review focused on the prognostic role of cytogenetics in Philadelphia (Ph)-negative myeloproliferative neoplasms (MPN) was recently published in Medicina.

In the article, the authors concentrated on the most prevalent Ph-negative MPN classifications: polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis.

Cytogenetics has a well-established prognostic role in acute leukemias and in myelodysplastic syndromes, where it drives clinical decisions, and the cytogenetics analysis in Ph-negative MPNs can similarly offer useful information on prognosis.

Although the natural histories of Ph-negative MPNs can sometimes last decades, the authors point out that over that time, altered DNA methylation, which is associated with age and mutations, can also cause DNA breakage that can lead to deletions and duplications. Along with acquired point mutations and telomere shortening, these chromosomal alterations are also prognostically important for leukemic transformation.

The conventional method for cytogenetics is G-banding of metaphase nuclei from a cell culture to obtain a karyotype. Additional techniques include fluorescence in situ hybridization with labeled DNA probes, and hybridization with protein nucleic acid probes.

Read more

According to a study in the the AJMC

Despite excellent prognoses in low-risk patients with essential thrombocytopenia (ET) and polycythemia vera (PV), knowledge gaps remain and novel, more tolerable therapies require reevaluation of treatment algorithms.

Learn more

A phase 2 study suggests that tipifarnib may be more beneficial for patients with CMML, MDS, or MPNs and RAS pathway mutations than the general population of patients with these disease. 

Tipifarnib (Zarnestra) demonstrated modest efficacy benefit in patients with chronic myelomonocytic leukemia (CMML) and other myelodysplastic (MDS)/myeloproliferative neoplasms (MPNs) and was reasonably well-tolerated, according to results from a phase 2 study (NCT02807272). As a potent and highly selective farnesyl transferase inhibitor, tipifarnib therapy can be administered to work against the RAS pathway mutation that occurs in about 30% of patients with CMML and other MDS and MPNs. These mutations include NRAS, KRAS, CBL, and PTPN11. Earlier results of tipifarnib treatment showed that the agent is especially efficacious in patients with RAS wild-type CMML and in the MDS/MPN population, having high CXCR4/CXCR2 may be predictive of tipifarnib’s activity.

A total of 44 patients were included in the study and treated with tipifarnib 400 mg orally twice daily on days 1–21 of 28-day cycles. The primary end point was objective response rate (ORR) assessed per the MDS/MPN International Working Group (IWG) criteria in CMML patients with KRAS-positive or NRAS wild-type disease, and in the MDS/MPN population with high and low CXCR4/CXCR2. Secondary end points explored in the study include the adverse event profile of tipifarnib, progression-free survival (PFS) at 1 year, overall survival (OS) at 1 year, and duration of response (DOR).

Learn more