On November 12, 2021 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported that initial clinical data from Part A of its DRAGON Phase 1 proof-of-concept trial (NCT04291079) at the ongoing 36th Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (November 10-14, 2021), which supported dose selection and advancement into Part B (Press release, Scholar Rock, NOV 12, 2021, View Source;L-1-for-the-Treatment-of-Solid-Tumors—-Announces-Advancement-into-Part-B [SID1234595472]). The DRAGON trial is investigating SRK-181, a selective inhibitor of TGFβ1 activation, in patients with locally advanced or metastatic solid tumors that have shown primary resistance to checkpoint inhibitor therapies.

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The main objectives of DRAGON Part A are to evaluate the safety and tolerability of SRK-181 alone (Part A1) or in combination with anti-PD-(L)1 checkpoint inhibitor therapy (Part A2) and to determine the recommended dose for the Part B dose expansion phase. Part A1 enrolled patients who have experienced treatment failure from available standard of care therapy, and Part A2 enrolled patients who did not respond to prior anti-PD-(L)1 therapy. Based on the safety and pharmacokinetic data from Part A, Scholar Rock has initiated Part B, which is evaluating SRK-181 dosed 1500 mg every three weeks (Q3W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q3W and 1000 mg every two weeks (Q2W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q2W.

"While checkpoint inhibitor therapy has definitely advanced the treatment of cancer, resistance to this type of therapy remains a very significant unmet medical need," said Nagesh Mahanthappa, Ph.D., Interim CEO. "We are excited that the DRAGON Part A results support moving forward in Part B with a dose of SRK-181 aimed at robustly suppressing TGFβ1 signaling, and look forward to advancing our program to test our hypothesis that SRK-181 can overcome resistance to checkpoint inhibitors thereby increasing the number of patients who may benefit from cancer immunotherapy."

Details for the virtual e-poster are as follows:

Title: First-in-Human Phase 1 Trial of SRK-181: A Latent TGFβ1 inhibitor, Alone or in Combination with Anti-PD-(L)1 Treatment in Patients with Advanced Solid Tumors (DRAGON trial) (#532). The full abstracts were made available on the SITC (Free SITC Whitepaper) website on November 9, 2021.
Time: Virtual e-posters will be on display on the SITC (Free SITC Whitepaper) 2021 virtual meeting platform starting November 12, 2021.
Highlights from the DRAGON Part A data presented at SITC (Free SITC Whitepaper) include:

As of September 7, 2021, 29 patients have been dosed in Part A of the trial. The median number of prior lines of therapy was 4 (range 1, 9) for Part A1 and 4 (range 2, 6) for Part A2.
As of October 12, 2021, no dose-limiting toxicities were observed with SRK-181 in Part A. Doses up to 3000 mg Q3W and 2000 mg Q2W as a monotherapy in Part A1 and 1600 mg Q3W in combination with anti-PD-(L)1 therapy in Part A2 have been evaluated.
The most common (>10%) treatment-emergent adverse events of any grade related to treatment were fatigue, decreased appetite, and nausea (Part A1) and rash maculo-papular (Part A2).
Preliminary data showed a pharmacokinetic (PK) profile of SRK-181 consistent with that which is generally observed for monoclonal antibodies.
Preliminary anti-tumor effects were assessed using RECIST1.1 and reported based upon local investigator reads:
Among 19 patients in Part A1 (monotherapy), eight patients had a best response of stable disease (SD). There were three patients with ovarian cancer in Part A1; each of these patients had stable disease, with tumor regression observed in two patients.
Among 10 patients in Part A2 (combination therapy), one patient with renal cell carcinoma (RCC) who had a lack of response to prior anti-PD-1 therapy had a partial response (PR) after treatment with SRK-181 dosed 800 mg Q3W in combination with the same anti-PD-1 therapy. In addition, four patients had a best response of SD.
The efficacy and safety of SRK-181 are being evaluated in DRAGON Part B. With early data anticipated to be available in 2022, Part B will enroll and dose patients in multiple proof of concept cohorts conducted in parallel, including urothelial carcinoma (UC), cutaneous melanoma (MEL), non-small cell lung cancer (NSCLC), as well as a miscellaneous cohort of other solid tumors. Another cohort focusing on patients with clear cell renal cell carcinoma (ccRCC) is being added based on emerging insights, including preliminary data from Part A. Each cohort will enroll up to 40 patients with locally advanced or metastatic solid tumors who have demonstrated primary resistance to anti-PD-(L)1 therapy. The ccRCC cohort will also explore the effects of SRK-181 in patients with relapsed response after anti-PD-(L)1 treatment. Patients in the UC, MEL, NSCLC and ccRCC cohorts will be treated with SRK-181 in combination with pembrolizumab, and patients in the miscellaneous solid tumor cohort will be treated with SRK-181 in combination with any approved anti-PD-(L)1 therapy.

The selection of the Part B dose was based upon safety and PK results from Part A. Patients receiving an approved anti-PD-(L)1 therapy dosed Q3W will be dosed with SRK-181 dosed 1500 mg Q3W, while patients receiving an approved anti-PD-(L)1 therapy dosed Q2W will be dosed with SRK-181 dosed 1000 mg Q2W. Drug exposures from these regimens are anticipated to exceed the levels hypothesized as needed for anti-tumor effects, as predicted from PK modeling and preclinical tumor model data.

"We are pleased by the Part A data, which have enabled the initiation of Part B with a dose regimen aimed at robustly testing the therapeutic hypothesis for SRK-181 in overcoming tumor resistance to anti-PD-(L)1 therapy," said Yung Chyung, M.D., CMO. "Part B has been specifically enriched with solid tumor types for which we believe there may be a higher potential for early efficacy signals on the basis of translational and preclinical insights."

About SRK-181
SRK-181 is a selective inhibitor of TGFβ1 activation and is an investigational product candidate being developed to overcome primary resistance to checkpoint inhibitor therapy, such as anti-PD-(L)1 antibodies. TGFβ1 is the predominant TGFβ isoform expressed in many human tumor types. Based on analyses of various human tumors that are resistant to anti-PD-(L)1 therapy, data suggest TGFβ1 is a key contributor to the immunosuppressive tumor microenvironment, excluding and preventing entry of cytotoxic T cells into the tumor, thereby inhibiting anti-tumor immunity (1). Scholar Rock believes SRK-181, which specifically targets the latent TGFβ1 isoform, has the potential to overcome this immune cell exclusion and induce tumor regression when administered in combination with anti-PD-(L)1 therapy while potentially avoiding toxicities associated with non-selective TGFβ inhibition. The DRAGON Phase 1 proof-of-concept clinical trial (NCT04291079) in patients with locally advanced or metastatic solid tumors is ongoing. The efficacy and safety of SRK-181 have not been established. SRK-181 has not been approved for any use by the FDA nor any other regulatory agency.

(1) Martin et al., Sci. Transl. Med. 12: 25 March 2020

On November 12, 2021 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported financial results for the third quarter ended September 30, 2021 and provided a clinical and corporate update (Press release, Milestone Pharmaceuticals, NOV 12, 2021, View Source [SID1234595488]).

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"We continue to make meaningful progress across our etripamil PSVT program. We’ve advanced our Phase 3 efficacy and safety studies and completed important patient research which enables a deeper understanding and characterization of the burden experienced by patients," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "Additionally, we look forward to presenting data from an analysis of heart rate in patients treated in the NODE-301 trial at the upcoming AHA Scientific Sessions 2021 meeting. We believe these data underscore the potential of etripamil to serve as an important intervention for patients with episodic cardiovascular conditions."

Recent Updates

Milestone Remains on Track to Report Topline Data from Pivotal Phase 3 RAPID Trial in the Second Half of 2022. As previously announced, enrollment continues in the ongoing pivotal Phase 3 RAPID trial of etripamil nasal spray in patients with paroxysmal supraventricular tachycardia (PSVT). Milestone is working closely with study investigators to support patient enrollment and continues to activate new centers. The Company remains on track to report topline data in the second half of 2022.

The RAPID trial, which is targeting a total of 180 adjudicated PSVT events, is expected to randomize approximately 500 patients 1:1 to receive either etripamil or placebo. To maximize the potential treatment effect of etripamil, patients will be directed to administer a repeat dose of study drug if they do not experience symptom relief within 10 minutes of the first study drug administration. The primary efficacy analysis for both the RAPID trial and the completed NODE-301 trial will be time to conversion of supraventricular tachycardia (SVT) over the first 30 minutes following initial study drug administration, with a target p-value of less than 0.05 for each trial. The RAPID and NODE-301 trials could potentially serve to fulfill the efficacy requirement for a future New Drug Application (NDA) for etripamil in patients with PSVT.

Heart Rate Data from NODE-301 Study to be Presented at the American Heart Association (AHA) Scientific Sessions 2021. New analyses on the impact of etripamil on heart rate in patients with PSVT, from the NODE-301 Study, will be presented at the upcoming AHA Scientific Sessions 2021 meeting. The presentation, titled "Etripamil Nasal Spray Reduces Heart Rate in Patients with Paroxysmal Supraventricular Tachycardia Prior to Conversion to Sinus Rhythm", will be featured during an ePoster session on November 14, 2021 at 11:00 a.m. ET.

Analysis of Large Longitudinal Patient Reported Outcome Market Research Study Establishes Disease Burden in PSVT and Market Opportunity for Etripamil. In the third quarter, Milestone completed an important patient reported outcomes (PRO) market research study in PSVT. The 247 patients who participated in the longitudinal portion of the study represent the broader PSVT population in terms of age, sex, medical history and time since diagnosis. Patients on average participated for 8.5 months and completed a survey every 12 days. In total, over 5,000 episodes were reported and characterized. Of these episodes, approximately 60% lasted longer than 10 minutes and 35% longer than 30 minutes.

Patients who participated in the study demonstrated a wide range of annual SVT episode frequency (0 to >50), with a median frequency of 12-15 episodes per year. Based on internal analysis, Milestone estimates approximately 60% of patients experience multiple 10+ minute episodes each year characterized as moderate or severe in intensity. In addition, approximately 30% of patients experiencing episodes sought medical care for the episode, the majority of which were treated in the emergency department.

ReVeRA Phase 2 Proof-of-Concept Trial Continues Recruitment in Patients Experiencing Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR). Recruitment is ongoing in ReVeRA, Milestone’s Phase 2 proof-of-concept study of etripamil nasal spray in patients experiencing AFib-RVR. Patients are being randomized 1:1 to receive either 70 mg of etripamil or placebo. The Phase 2 double blind, placebo controlled, proof-of-concept in-patient study is designed to assess the safety and efficacy of etripamil nasal spray to reduce the ventricular rate in patients with AFib-RVR. The trial is being conducted in Canada in collaboration with the Montreal Heart Institute and other research centers. The primary endpoint will assess reduction in ventricular rate, with key secondary endpoints including the time to achieve the maximum reduction in rate and duration of the effect.
Third Quarter 2021 Financial Results

As of September 30, 2021, Milestone had cash, cash equivalents, and short-term investments of $126.4 million and 29.9 million common shares issued and outstanding and 12.3 million common shares issuable upon exercise of pre-funded warrants outstanding.

Research and development expense for the third quarter of 2021 was $9.7 million compared with $8.2 million for the prior year period. The increase reflects higher clinical consulting fees and contract research organization (CRO) costs due to advancing RAPID Phase 3 efficacy and safety trials in etripamil for the treatment of PSVT along with an increase in clinical personnel related costs. For the nine months ended September 30, 2021, research and development expense was $27.8 million compared with $28.7 million for the prior year period. The decrease was due to a reduction in clinical trial expenses of $2.3 million which was partially offset by an increase of $1.3 million in clinical personnel related costs which included a non-cash share-based compensation expense.

General and administrative expenses for the third quarter of 2021 and 2020 were $3.0 million. For both of the nine month periods ended September 30, 2021 and 2020, respectively, general and administrative expense was $8.6 million.

Commercial expense for the third quarter of 2021 was $1.6 million compared with $0.9 million for the prior year period. The increase is attributable to investment in commercial activities during the three months ended September 30, 2021 in contrast to the three months ended September 30, 2020, a period during which Milestone reduced commercial spending in order to focus efforts on an optimized clinical development pathway for etripamil after issuing topline results of the first part of the NODE-301 in March 2020. For the nine months ended September 30, 2021, commercial expense was $4.8 million compared with $4.6 million for the prior year period. This change was due to an increased investment in commercialization activities.

For the third quarter of 2021, operating loss was $14.3 million compared to $12.1 million in 2020. For the nine months ended September 30, 2021, Milestone’s operating loss was $26.2 million compared to $41.9 million in the prior year period.
About Paroxysmal Supraventricular Tachycardia

Paroxysmal supraventricular tachycardia (PSVT) is a condition characterized by intermittent episodes of rapid heart beat that starts and stops suddenly and without warning that affects approximately two million Americans. Episodes of supraventricular tachycardia (SVT) are often associated with palpitations, sweating, chest pressure or pain, shortness of breath, sudden onset of fatigue, lightheadedness or dizziness, fainting, and anxiety. Adenosine and certain calcium channel blockers have long been approved for the treatment of PSVT. However, these medications must be administered intravenously under medical supervision, usually in an emergency department or other acute care setting.

About Atrial Fibrillation with Rapid Ventricular Rate

Atrial fibrillation (AFib) is a common arrhythmia marked by an irregular and often rapid heartbeat. AFib is estimated to affect five million patients in the United States, a prevalence projected by the Centers for Disease Control to increase to twelve million patients within the next 10 years. Atrial fibrillation with rapid ventricular rate (AFib-RVR) is a condition in which patients with AFib experience episodes of abnormally high heart rate, often with symptoms such as palpitations, shortness of breath, dizziness, and weakness. Oral calcium channel blockers and/or beta blockers are commonly used to manage the heart rate in this condition. When episodes do occur, the corresponding symptoms often cause patients to seek care in the acute care setting such as the emergency department, where standard of care procedures include intravenous administration of calcium channel blockers or beta blockers under medical supervision. Milestone’s initial qualitative market research indicates that approximately 40% of patients with AFib experience one or more symptomatic episodes of RVR per year that require treatment, suggesting a target addressable market for etripamil in patients with AFib of approximately two million patients.

About Etripamil

Etripamil, Milestone’s lead investigational product, is a novel calcium channel blocker designed to be a rapid-response therapy for episodic cardiovascular conditions. As a nasal spray that is self-administered by the patient, etripamil has the potential to shift the current treatment experience for many patients from the emergency department to the at-home setting. Milestone is conducting a comprehensive development program for etripamil, with Phase 3 trials ongoing in paroxysmal supraventricular tachycardia (PSVT) and now a Phase 2 proof-of-concept trial underway in patients with atrial fibrillation and rapid ventricular rate (AFib-RVR).

On November 12, 2021 Upsher-Smith Laboratories reported that (Press release, Upsher-Smith Laboratories, NOV 12, 2021, View Source [SID1234595511])

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Japan: Despite the impact of the NHI drug price revision, both sales and core operating income exceeded the same period of the previous fiscal year due to increased sales of products launched in FY2020 and increased demand for Sawai Pharmaceutical products due to the supply stoppages etc. of products from other generics companies.

 United States: Both sales and core operating income decreased from the same period last year, due to the impact of competitor entries into key USL generic product markets

 Sales declined significantly, due to the impact of competitor entries into key USL generic product markets.
 Steady progress in brand products.
 Three products have launched in the first half of FY2021.

Combined performance for Zembrace and Tosymra is ahead of expectations for FY21; Tosymra volumes are in line with plan while Revenue is slightly behind plan due to lower than planned average selling price (ASP)

▍Key Actions to Accelerate Tosymra Growth
Drive improved Average Selling Price (ASP) through improved Prior Authorization (PA) performance (both submission rates and success rates for submitted PAs) and new Platinum Pass business rules

Drive new prescription growth via new Direct-to-Consumer channel partnerships with Cove, an online migraine telemedicine company, and two regional pharmacies to conduct patient consultations on non-oral migraine solutions
(Tosymra)

Engage new managed care partner, Eversana, and leverage real world economic data to influence better coverage from managed care

Continue to drive first fill success through specialty pharmacy channel with Blink Pharmacy

 The business in Japan grew steadily, while the sales in the US fell short of the initial forecast due to a sharp decline
in sales of main generics products.
 On a consolidated basis, both net sales and core operating income slightly exceeded the FY2021 first half forecast.
 Our full-year forecast remains unchanged as the circumstances surrounding the business environment are unclear,
considering shipment adjustments due to the supply stoppages of products from other generics companies etc

On November 12, 2021 Kenjockety Biotechnology, Inc., an early-stage biotechnology company focused on discovery and development of novel therapeutics for the treatment of patients with drug-resistant cancers, reported new data for their lead BsAb program targeting Pgp and CD47 (Press release, Kenjockety Biotechnology, NOV 12, 2021, View Source [SID1234595527]). These data will be shared in a poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting in Washington, DC.

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"These results demonstrate proof-of-concept for our lead bispecific antibody which is now ready for IND enabling studies," said Robert Arathoon, PhD, Founder and CEO of Kenjockety Biotechnology. "With this novel approach we engineer bispecific antibodies to precisely target tumor but not normal cells, enabling enhanced efficacy while reducing toxicities seen with small molecules or monoclonal antibodies."

The presentation includes preclinical results demonstrating in vivo efficacy for a Pgp X CD47 BsAb, both as monotherapy and in combination with paclitaxel in multiple tumor models. Kenjockety’s unique approach enables a multi-modal mechanism of action. This is achieved by antagonizing functional aspects of both targets: inhibiting Pgp to reduce drug resistance while targeting CD47 to enhance immune attack. Additionally, Kenjockety’s results illustrate tumor-specific targeting by engineering BsAbs to have strong binding only in the presence of both targets.

"Our proprietary BASE Platform facilitates the design and engineering of new therapeutics that antagonize Efflux Pumps, like Pgp, together with immuno-oncology targets, such as CD47 or other TAAs," said Dr. Arathoon. "These therapeutics have the potential to provide significant clinical benefits for patients with a wide array of cancers."

Presentation Information

Title: Bispecific Antibodies (BsAbs) Targeting ABCB1/P-Glycoprotein (Pgp) and CD47 Provide a Multimodal, Tumor-Specific Approach to Combat Drug-Resistant Cancers

Abstract Number: 282

Date/Time: Saturday, November 13th, 7:00 a.m. – 8:30 p.m.

Session: Poster and Exhibit Hall

Presenter: Dr. Robert Arathoon, Founder and CEO, Kenjockety Biotechnology, Inc.

On November 12, 2021 Biosight Ltd., a pharmaceutical development company developing innovative therapeutics for hematological malignancies and disorders, reported the initiation of a Phase 2 trial to evaluate aspacytarabine (BST-236), Biosight’s proprietary antimetabolite, as a second line treatment for patients with relapsed or refractory myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (Press release, Advaxis, NOV 12, 2021, View Source;soc_trk=ma [SID1234595728]). The multi-center study will be conducted across 18 leading U.S. and Israeli sites including Memorial Sloan Kettering Cancer Center and The University of Texas MD Anderson Cancer Center.

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"Biosight has built a strong foundation of compelling data that suggests that aspacytarabine may serve as a more tolerable and effective standard of care treatment for patients with AML," said Dr. Ruth Ben Yakar, Chief Executive Officer of Biosight. "The initiation of this multi-center, U.S. based, Phase 2 study is an important step forward in the development of aspacytarabine, seeking to address unmet needs in the treatment of patients with relapsed or refractory AML and MDS. We are proud to be continuing our momentum in the clinic and look forward to collaborating with leading academic medical centers as we progress the study."

Eytan Stein, M.D., Hematologic Oncologist at Memorial Sloan Kettering Cancer Center and lead investigator of the study said "I’m encouraged by the results from Biosight’s Phase 2b trial to be presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The efficacy achieved in a challenging population, across key measures including complete remission and MRD (-) rates, duration of response and overall survival, are noteworthy. Furthermore, these results were particularly impressive as they were achieved with a favorable safety and tolerability profile in patients who are unfit for intensive chemotherapy. Patients with relapsed or refractory AML and MDS have limited treatment options and poor prognoses, with many patients unable to tolerate intensive chemotherapy. I look forward to leading this new Phase 2 study to evaluate the potential of aspacytarabine which may ultimately become the standard of care for relapsed or refractory MDS and AML patients for whom currently there are no effective treatments."

The Phase 2 open label multi-center study will assess the safety and efficacy of BST-236 as a single agent in adult patients unfit for standard therapy with AML or higher-risk (HR)MDS who fail to respond to, or have relapsed following, first line therapy. A similar study in patients with relapsed/refractory AML and MDS is ongoing in collaboration with the European cooperative group, Groupe Francophone des Myélodysplasies (GFM). Approximately 40 adult patients with relapsed and/or refractory AML and approximately 40 adult patients with relapsed and/or refractory HR MDS will be enrolled into the two studies. Primary endpoints include complete remission (CR) rate in AML patients and Overall Response Rate (ORR) in MDS patients, with ORR defined as the proportion of patients who achieve a CR or partial response (PR) per proposal for modification of the International Working Group (IWG) criteria for MDS, 2006.

About Aspacytarabine (BST-236)

Aspacytarabine is a novel proprietary anti-metabolite. It is composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine. Cytarabine has served as the backbone of AML therapy for over 45 years due to its superior efficacy. However, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically compromised patients. Due to its unique pharmacokinetics and metabolism, aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues. As such, aspacytarabine may serve as a new therapy for AML and other hematological malignancies and disorders, including for older adults who are unfit for intensive therapy.

Aspacytarabine was granted FDA Fast Track Designation for treatment of AML patients unfit for standard chemotherapy, and FDA and EMA Orphan Drug Designations, which entitle Biosight to seven and ten years of market exclusivity in the U.S. and Europe, respectively, upon aspacytarabine marketing approval for the treatment of AML in each territory.

Interim results from an ongoing Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and single-agent activity, and additional studies are ongoing to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory MDS or AML. For more information regarding the Phase 2b clinical study of BST-236, please visit www.clinicaltrials.gov.