On May 13, 2021 Systems Oncology, a biotechnology company, reported that Dr. Spyro Mousses, CEO and Co-Founder of Systems Oncology, will participate in a fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference on Thursday, May 20th at 12:40pm EST (Press release, Systems Oncology, MAY 13, 2021, View Source [SID1234579956]).

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Management will host one-on-one investor meetings at the conference. To request a meeting with management, please contact your registered RBC representative or Andrew Matricaria, CFO of Systems Oncology, at [email protected].

On May 13, 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 financial results for the first quarter ended March 31, 2021 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAY 13, 2021, View Source [SID1234579955]).

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Company Updates and Upcoming Milestones

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

TOPAZ 12-Month Data Show Transformative Potential in SMA of Apitegromab as Add-on to SMN Upregulator Therapy. In April 2021, positive top-line data were announced from the TOPAZ Phase 2 trial (NCT03921528) in patients with Type 2 and Type 3 SMA. Both younger and older non-ambulatory patients treated with apitegromab and background SMN upregulator therapy over the 12-month period attained meaningful motor function improvements with 74% (23/31) of patients demonstrating a clinical improvement of ≥1-point increase in Hammersmith Functional Motor Scale Expanded (HFMSE).

In the younger non-ambulatory TOPAZ cohort (ages 2-6 years), treatment with apitegromab led to a mean increase from baseline in HFMSE of +6.2 points across both evaluated doses; a mean increase of +7.1 points for the 20 mg/kg dose and +5.3 points with the 2 mg/kg dose.

Increase in HFMSE from baseline across both apitegromab doses:

− 82% (14/17) of patients attained a >1-point increase
− 59% (10/17) of patients attained a >3-point increase
− 59% (10/17) of patients attained a >5-point increase
− 35% (6/17) of patients attained a >10-point increase

In the older non-ambulatory TOPAZ cohort (age 8-19 years), treatment with apitegromab 20 mg/kg led to a mean increase from baseline in HFMSE of +0.6 points by the primary intent-to-treat analysis and +1.2 points by the prespecified per protocol analysis.

Increase in HFMSE from baseline for this patient population:

− 64% (9/14) of patients attained a >1-point increase
− 29% (4/14) of patients attained a >3-point increase
Patients enrolled in TOPAZ were receiving chronic maintenance doses of nusinersen; both the younger and older non-ambulatory cohorts had received more than five mean maintenance doses of nusinersen (approximately two years of treatment) at baseline. Clinical data from the CHERISH and SHINE studies of nusinersen offer background insights into this patient population.1 These studies observed that nusinersen-treated patients primarily experience stabilization or only slight increases in HFMSE scores beyond the initial 15-month treatment period.2 In addition, even in the initial 15-month treatment period, older patients (age 5-12) on average experience declines in HFMSE and rarely attain a >3-point increase in HFMSE in a 12-month timeframe.3

The five most frequently reported treatment-emergent adverse events included headache, pyrexia, upper respiratory tract infection, cough, and nasopharyngitis​. All 57 patients who completed the 12-month treatment period elected to opt into the extension period.

Phase 3 Registrational Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Anticipated to Initiate by Year-End 2021. TOPAZ Phase 2 top-line efficacy and safety results support the continued evaluation of apitegromab in a Phase 3 trial. Subject to feedback from regulatory agencies, Scholar Rock intends to conduct a Phase 3 trial to evaluate apitegromab with background SMN upregulator therapy in non-ambulatory Type 2 and Type 3 SMA, the patient population where apitegromab demonstrated the largest increases in motor function (HFMSE scores) in TOPAZ. Patients with non-ambulatory Type 2 and Type 3 SMA represent approximately two-thirds of the overall prevalent SMA patient population.
Additional Data and Analyses from the TOPAZ Trial to be Presented at Upcoming Medical Congresses. Scholar Rock is conducting additional data analyses, including exploratory analyses using patient-level data, evaluating additional outcome measures, and reviewing additional safety data from the TOPAZ Phase 2 trial. The Company plans to present the TOPAZ top-line results as well as findings from these analyses at upcoming medical congresses.
Apitegromab Granted Priority Medicines (PRIME) Designation by the European Medicines Agency (EMA). In March 2021, the EMA granted PRIME designation to apitegromab, recognizing its potential to address unmet medical needs of patients with SMA. With the PRIME designation, Scholar Rock has greater access and enhanced dialogue with the regulatory agency to optimize development plans and evaluations.
Two U.S. Patents Issued Adding Further Protection for Inhibitors of Myostatin Activation and Related Methods, Including Apitegromab. In March 2021, the United States Patent Office (USPTO) issued U.S. Patent No. 10,946,036 with an expiry of June 2037, protecting both add-on and combination therapy with a myostatin inhibitor and a neuronal corrector therapy (such as SMN upregulator therapy) for the treatment of SMA. In April 2021, the USPTO issued U.S. Patent No. 10,981,981 with an expiry of May 2034, broadly covering methods for making inhibitors of myostatin (GDF8) activation based on Scholar Rock’s proprietary platform approach of targeting the precursor forms of growth factors.
Additional Potential Indications Identified for Apitegromab. Scholar Rock has identified multiple diseases for which the selective inhibition of the activation of myostatin may offer therapeutic benefit, including additional patient populations in SMA (such as Type 1 SMA and ambulatory Type 3 SMA) and potential indications outside of SMA, such as Becker Muscular Dystrophy (BMD).
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Published Preclinical Development Data for SRK-181 in International Journal of Toxicology. In March 2021, Scholar Rock announced the publication of "Nonclinical Development of SRK-181: An Anti-latent TGFβ1 Monoclonal Antibody for the Treatment of Locally Advanced or Metastatic Solid Tumors," in the peer-reviewed International Journal of Toxicology. This publication provided a comprehensive preclinical assessment of the pharmacology, pharmacokinetics, and safety of SRK-181, which provided support for the dose selection strategy for the ongoing DRAGON Phase 1 trial.
Initial Clinical Response and Safety Data from Part A of the DRAGON Phase 1 Trial Anticipated by Year-End 2021. SRK-181 is being evaluated in the two-part DRAGON trial (NCT04291079) in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Dose escalation continues to progress in Part A of the trial, which evaluates the safety and pharmacokinetics of SRK-181, and the Company expects to advance to the Part B dose expansion portion of the trial in mid-2021. Part B will consist of multiple cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Each cohort will enroll up to 40 patients who have demonstrated primary resistance to anti-PD-(L)1 therapy and will be treated with SRK-181 in combination with an approved anti-PD-(L)1 therapy.
"2021 is off to a great start as we are delighted with the positive 12-month top-line data from our TOPAZ study, which shows the transformative potential of apitegromab in SMA," said Tony Kingsley, President and CEO of Scholar Rock. "Based on these exciting results and the potential to bring apitegromab to the SMA community, we will be working with urgency on behalf of patients to initiate our Phase 3 registrational trial in patients with non-ambulatory Type 2 and 3 SMA by the end of the year."

First Quarter 2021 Financial Results

For the quarter ended March 31, 2021, net loss was $27.7 million or $0.76 per share compared to a net loss of $17.1 million or $0.58 per share for the quarter ended March 31, 2020.

Revenue was $4.7 million for the quarter ended March 31, 2021 compared to $5.0 million for the quarter ended March 31, 2020 and was related to the Gilead fibrosis-focused collaboration (the "Gilead Collaboration Agreement") that was executed in December 2018.
Research and development expense was $22.5 million for the quarter ended March 31, 2021 compared to $16.9 million for the quarter ended March 31, 2020. The increase year-over-year primarily reflects manufacturing costs associated with apitegromab and higher personnel and facility-related costs, partially offset by lower manufacturing costs associated with SRK-181.
General and administrative expense was $9.4 million for the quarter ended March 31, 2021 compared to $5.8 million for the quarter ended March 31, 2020. The increase year-over-year was primarily attributed to higher personnel and facility-related costs.
"We are focused on advancing our portfolio of clinical and preclinical programs with several near-term milestones, including the initiation of a Phase 3 trial evaluating apitegromab in SMA, completion of dose escalation in Part A and initiation of the Part B dose expansion portion of the DRAGON Phase 1 trial of SRK-181 in patients with solid tumors," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We are well positioned to continue to execute on our plans as we ended the first quarter with approximately $315 million in cash, which can fund operations into 2023."

1This information from third-party studies is provided for background purposes only and is not intended to convey or imply a comparison to the TOPAZ clinical trial results.
2 Source: "Longer-term treatment with nusinersen: results in later-onset spinal muscular atrophy from the SHINE study" P.257, World Muscle Society Congress 2020
3 Source: Mercuri E, et.al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378:625-635.

On May 14, 2021 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Selecta Biosciences, MAY 13, 2021, View Source [SID1234579954]).

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"We are very pleased about the continued progress across all aspects of the company," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "We regained exclusive rights to our MMA program and now have two proprietary gene therapy programs to rapidly follow our ongoing empty capsid study. Additionally, we continue to progress our enzyme program, with an expected IND filing by the end of 2021 in IgA nephropathy, and topline data from the Phase 3 DISSOLVE program for SEL-212 anticipated in the second half of 2022."

Recent Highlights and Anticipated Upcoming Milestones:

Enzyme Therapies:

SEL-212 for chronic refractory gout: Enrollment for the Phase 3 DISSOLVE clinical program for SEL-212 for the treatment of chronic refractory gout, which was licensed to Sobi, is progressing as planned with topline data expected in the second half of 2022.
Leveraging the success of SEL-212, Selecta expects to file an Investigational New Drug, or IND, application by the end of 2021 for a novel therapeutic approach that combines ImmTOR with an enzyme, IgA1 protease for the treatment of IgA nephropathy.
Gene Therapies:

First-in-human trial of SEL-399: In collaboration with AskBio, Selecta initiated the first-in-human, dose-escalation trial of SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) containing no DNA combined with ImmTOR. The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies. Selecta and AskBio expect to report topline data in the fourth quarter of 2021.
MMA-101 for methylmalonic acidemia (MMA): Selecta regained exclusive rights to its lead gene therapy program in MMA from AskBio and expects to file an IND in MMA-101, in combination with ImmTOR, by the end of 2021. The phase 1/2 MMA-101 program, which is expected to commence in 2022, will evaluate biomarkers of efficacy, neutralizing antibodies and safety and tolerability.
SEL-313 for ornithine transcarbamylase deficiency (OTC deficiency): Selecta’s proprietary gene therapy product candidate, SEL-313, is being developed to treat OTC deficiency, a rare genetic urea cycle disorder that causes ammonia to accumulate in the blood due to mutations in the OTC gene. SEL-313 is currently in preclinical development and a clinical trial application, or CTA and/or IND filing are expected in 2022. A Pediatric Investigation Plan (PIP) for SEL-313 was submitted to the European Medicines Agency (EMA) pediatric committee in February 2021.
Sarepta Therapeutics program in Duchenne Muscular Dystrophy (DMD) and certain Limb-Girdle Muscular Dystrophies (LGMD) subtypes: Selecta has achieved a $3 million milestone payment related to the completion of a preclinical study under the Research License and Option Agreement.
Restoring Self-Tolerance in Autoimmune Diseases:

Selecta continues IND-enabling work on an ImmTOR-based approach to treating primary biliary cholangitis (PBC), a chronic, progressive autoimmune liver disorder that leads to inflammation, damage and scarring of the small bile ducts. Selecta expects to file an IND in PBC in the second half of 2022.
Corporate Updates:

Kristen Baldwin was appointed Chief People Officer. She brings 20 years of Human Resources and Consulting experience to the company. Most recently Ms. Baldwin served in dual capacity as the Chief People Officer for the LIVEKINDLY Collective, a high growth plant-based foods company, and as a Senior Partner at CEO.works. Ms. Baldwin has also held senior HR roles at Bayer and Otsuka Pharmaceuticals.

Satish Tripathi, Ph.D., was appointed Vice President of Global Regulatory Affairs. Dr. Tripathi has over 25 years of combined R&D, business, and global regulatory strategy experience. Dr. Tripathi most recently served as VP of Global Regulatory Affairs for AveXis which became Novartis Gene Therapies, where he led the regulatory strategy and implementation for the gene therapy product AVXS-101 for Spinal Muscular Atrophy. AVXS-101 is recognized as only one of the 3 drugs in the world to receive Breakthrough (US FDA), PRIME (EMA) and Sakigake (MHLW/PMDA) designations. Dr. Tripathi led the simultaneous submission of AVXS-101 in 2018 for global registration, which has been approved as Zolgensma for SMA in US, Europe, Japan, Canada, and Brazil.

Brad Dahms will be stepping down as Chief Financial Officer effective May 21st, 2021 to pursue another opportunity. Mr. Dahms’ departure is not related to Selecta’s operations, financial reporting, or controls. A search is currently underway for a successor.

Ann Donohue will be promoted to Vice President Finance, effective immediately, after having served as Controller of Selecta since December 2017.
First Quarter 2021 Financial Results:

Cash Position: Selecta had $149.2 million in cash, cash equivalents, marketable securities, and restricted cash as of March 31, 2021, which compares to cash, cash equivalents, and restricted cash of $140.1 million as of December 31, 2020. Selecta believes its available cash, cash equivalents, marketable securities, and restricted cash will be sufficient to meet its operating requirements into the second quarter of 2023.

Net cash used in operating activities was $12.1 million for the first quarter of 2021, as compared to $11.7 million for the same period in 2020.
Revenue: Revenue recognition for the first quarter of 2021 was $11.1 million, compared to no revenue recognition for the same period in 2020. Revenue was recognized under the license agreement with Sobi which began in July 2020 resulting from the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program.

Research and Development Expenses: Research and development expenses for the first quarter 2021 were $13.0 million, which compares with $14.7 million for the same period in 2020. During the quarter ended March 31, 2021, there was a reduction in expenses for the SEL-212 clinical program and for the AskBio Collaboration, offset by an increase of expense for discovery and preclinical programs.

General and Administrative Expenses: General and administrative expenses for the first quarter 2021 were $5.2 million, which compares with $4.1 million for the same period in 2020. The quarterly increase in expense was the result of expenses for consulting and professional fees and salaries offset by reduced travel expenses.

Net Loss: For the first quarter 2021, Selecta reported a net loss of $24.6 million, or $0.22 per share, compared to a net loss of $19.6 million, or $0.21 per share for the same period in 2020.

Conference Call and Webcast Reminder:
Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s first quarter 2021 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10147801. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

On May 13, 2021 Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported new collaborations in Israel, Italy and Spain to expand molecular screening opportunities for patients with cancers that may have neuregulin 1 (NRG1) fusions and to raise awareness of the Merus eNRGy clinical trial of its bispecific antibody zenocutuzumab (Zeno) (Press release, Merus, MAY 13, 2021, View Source [SID1234579953]). In the collaborations, Merus plans to support molecular screenings for eligible patients with pancreatic adenocarcinoma in Israel and Italy, and with non-small cell lung cancer (NSCLC) in Spain, aimed to identify the presence of NRG1 fusions. Each collaborating organization in turn has agreed as follows:

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Progenetics, Ltd. plans to perform a nationwide campaign in Israel to raise awareness of the molecular screening offered by Merus for eligible pancreatic adenocarcinoma patients and availability of the eNRGy trial for eligible patients. Progenetics is a leading Israeli company in oncology testing, currently distributing diagnostic tests for nine American companies in the field of onco-diagnostics.

Italian Association for the Study of Pancreas (AISP) plans to inform their nationwide network of oncologists, pancreatic cancer patients and patient associations in Italy of the molecular screening offered by Merus for eligible pancreatic adenocarcinoma patients and availability of the eNRGy trial for eligible patients.

Dr. Michele Reni, a Principal Investigator on the eNRGy trial and physician at the San Raffaele Scientific Institute in Milan, has agreed to facilitate the collaboration with AISP and said, "Our collaboration with Merus, and the testing they are supporting, offers a unique opportunity to our patients and to physicians involved in the treatment of pancreatic cancer to help patients know their NRG1 fusion status and to potentially match patients with clinical trial opportunities that specifically target their unique tumor profiles."
Universidad de Navarra plans to provide Merus-funded molecular screening to eligible patients with NSCLC, through the Clínica Universidad de Navarra network in Spain, which may identify NRG1 fusions, and plans to inform patients with NRG1-fusion-positive cancer of their potential eligibility for the eNRGy trial. The Clínica Universidad de Navarra, based in Pamplona and Madrid, is a leading research hospital in Spain. A recognized institution for both its teaching and research work, and its trajectory in the diagnosis and treatment of highly complex pathologies, the Clínica Universidad de Navarra is characterized by the diagnostic speed achieved through multidisciplinary work and the acquisition of the latest technology to offer care in 46 different medical and surgical specialties.
"These latest collaborations expand Merus’ global efforts to raise awareness of the importance of molecular testing which can lead to potentially better treatment and clinical trial options for cancer patients," said Dr. Andrew Joe, Chief Medical Officer of Merus. "Merus’ support in the screening of these patients is also a strategic effort to potentially identify and recruit patients with cancer harboring NRG1 fusions for our eNRGy trial."

Merus has implemented a global approach designed to increase access to molecular screenings for cancer patients and to potentially enhance enrollment in the eNRGy trial by working with private industry, country-specific testing organizations, cooperative groups and disease-specific cancer organizations. Increasing access to molecular screenings may help oncologists and their patients, whose cancers may not be screened routinely for gene mutations, make informed decisions on what treatment and clinical trial options may be available.

With the addition of these collaborations announced today, Merus is now working with more than ten different industry and academic collaborators across Asia, North America and Europe aimed to enhance testing for NRG1 fusions and to raise awareness of the eNRGy trial.

About the eNRGy Clinical Trial
Merus is currently enrolling patients in the phase 1/2 eNRGy trial to assess the safety and anti-tumor activity of zenocutuzumab (Zeno) monotherapy in NRG1+ cancers. The eNRGy trial consists of three cohorts: NRG1+ pancreatic cancer; NRG1+ non-small cell lung cancer; and NRG1+ other solid tumors. Further details, including current trial sites, can be found at www.ClinicalTrials.gov and Merus’ trial website at www.nrg1.com or by calling 1-833-NRG-1234.

About NRG1 Fusions
The NRG1 gene encodes neuregulin (also known as heregulin), the ligand for HER3. Fusions between NRG1 and partner genes are rare, tumorigenic genomic events occurring in patients with certain cancers.

About Zeno
Zenocutuzumab (Zeno) is an antibody-dependent cell-mediated cytotoxicity (ADCC)-enhanced Biclonics that utilizes the Merus Dock & Block mechanism to inhibit the neuregulin/HER3 tumor-signaling pathway in solid tumors with NRG1 gene fusions (NRG1+). Through its unique mechanism of binding to HER2 and potently blocking the interaction of HER3 with its ligand NRG1 or NRG1-fusion proteins, Zeno has the potential to be particularly effective against NRG1+ cancers. In preclinical studies, Zeno also potently inhibits HER2/HER3 heterodimer formation and tumor growth in models harboring NRG1 fusions. Learn more about Zeno Dock & Block at View Source

On May 13, 2021 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported its participation in the Cowen 2nd Annual Virtual Oncology Innovation Summit. Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present in a virtual fireside chat on Thursday, May 20, 2021 at 10:20 a.m. PT / 1:20 p.m. ET (Press release, Kura Oncology, MAY 13, 2021, View Source [SID1234579951]).

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A live audio webcast of the presentation will be available in the Investors section of Kura’s website at www.kuraoncology.com, with a replay available shortly after the live event.