On March 7, 2022 aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways, reported that it will report fourth quarter and full year 2021 financial results and provide a corporate update after the market close on Monday, March 14, 2022 (Press release, aTyr Pharma, MAR 7, 2022, https://investors.atyrpharma.com/news-releases/news-release-details/atyr-pharma-webcast-conference-call-reporting-fourth-quarter-0 [SID1234609605]). Management will host a conference call and webcast to review the results and provide an operational update.

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On March 7, 2022 Harpoon Therapeutics, Inc. (NASDAQ: HARP), a clinical-stage immunotherapy company developing novel T cell engagers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN328, a delta like ligand 3- (DLL3) targeting TriTAC, for the treatment of small cell lung cancer (SCLC) (Press release, Harpoon Therapeutics, MAR 7, 2022, View Source [SID1234609604]). A Phase 1/2 clinical trial is currently ongoing for HPN328 in the SCLC patient population.

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"Orphan Drug Designation for HPN328 is a significant milestone that underscores the need for additional treatments for patients suffering from small cell lung cancer and HPN328’s potential to contribute to this unmet medical need," stated Julie Eastland, President and CEO, Harpoon Therapeutics. "We are pleased with the clinical progress of HPN328 and remain focused on dose escalation with the goal to determine the recommended Phase 2 dose by the end of this year."

The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and potential eligibility for seven-year marketing exclusivity upon FDA approval.

About the Phase 1/2 Trial for HPN328

HPN328 is a TriTAC that binds to human and non-human primate DLL3, CD3ε, and albumin with similar affinities. The Phase 1/2 trial is an open-label study of HPN328 as monotherapy to assess the safety, tolerability and pharmacokinetics in patients with advanced cancers associated with expression of DLL3. The first part of the trial is designed to determine a dose for additional clinical investigations.

As of the December 13, 2021 clinical update provided by Harpoon, 15 patients had been enrolled in dose cohorts ranging from 15 µg to 7200 µg per week using both fixed and step dose administration once weekly by intravenous infusion. Enrolled patients had a median of 2 lines (range 1 to 5) of prior therapy and included small cell lung cancer patients who had relapsed after platinum chemotherapy and patients with other malignancies with high grade neuroendocrine tumors associated with DLL3 expression. HPN328 has been well tolerated with Grade 1-2 cytokine release syndrome (CRS) reported in 33% of patients, no DLTs observed and MTD had not been reached. Among four patients with small cell lung cancer receiving the two highest doses tested to date, 1215 µg fixed dose and 3600-7200 µg step dose, three had target lesion reduction, including 1 confirmed RECIST partial response. The patient with a cPR experienced a target lesion reduction of 53% at week 10.

Following dose escalation, Harpoon may further evaluate the safety and efficacy of HPN328 in additional parallel cohorts. The primary outcome measure will be to determine efficacy for the Phase 2 dose based on the overall response rate as determined by RECIST. For additional information about the trial, please visit clinicaltrials.gov using the identifier NCT04471727.

On March 7, 2022 Oasmia Pharmaceutical’s CEO, Dr Francois Martelet reported that it will present at Aktiespararna’s Aktiedagen Stockholm on March 14, 2022 (Press release, Oasmia, MAR 7, 2022, View Source [SID1234609602]). The presentation starts at 11:00 CET and will be broadcast live as a webcast at: www.aktiespararna.se/tv/live.

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The presentation will be available online the following day on www.aktiespararna.se/tv/evenemang and on Oasmia Pharmaceutical’s website www.oasmia.com.

On March 7, 2022 Instil Bio, Inc. ("Instil") (NASDAQ: TIL), a clinical-stage biopharmaceutical company focused on developing tumor infiltrating lymphocyte, or TIL, therapies for the treatment of patients with cancer, reported its fourth quarter and year end 2021 financial results and provided a corporate update (Press release, Instil Bio, MAR 7, 2022, View Source [SID1234609601]).

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"I am pleased with our progress in establishing our operational capabilities and initiating a clinical study in our first year as a public company," said Bronson Crouch, Chief Executive Officer of Instil. "With the DELTA-1 Phase 2 trial of ITIL-168 underway and continued expansion of our manufacturing capabilities in both the United Kingdom and the United States, we continue to develop our platform to deliver cell therapies to patients. In 2022, we look forward to the opportunity to expand the targeted patient population for receiving our therapies in multiple indications with ITIL-168 and ITIL-306."

2021 Highlights and Anticipated Milestones:

Enrollment Ongoing in DELTA-1: Instil is enrolling patients in DELTA-1, a global Phase 2 clinical trial of ITIL-168 with registrational intent in patients with advanced melanoma whose disease has progressed following PD-1 inhibitor therapy and, if BRAF-mutated, targeted therapy. Instil expects top-line safety and efficacy data in 2023 which could potentially support BLA submission and a European Medicines Agency marketing authorization application (MAA) filing.

INDa Cleared for DELTA-2: Instil reported that in Q4 2021 the IND amendment was cleared for DELTA-2, a Phase 1 study of ITIL-168 in non-small cell lung cancer (NSCLC), head and neck squamous cell carcinoma (HNSCC), cervical cancer, and cutaneous squamous cell carcinoma (CSCC).

Readiness to Initiate Phase 1 Study of ITIL-306 in 2022: Instil remains on track to file an IND for a Phase 1 study of its first genetically engineered CoStAR-TIL, ITIL-306, in multiple solid tumor indications in H1 2022.

Presenting Pre-clinical Data on the CoStAR Platform: Instil expects to present further pre-clinical data on the CoStAR platform, including manufacturability and in vivo models of efficacy and safety, at an upcoming scientific conference in 2022. In November 2021, Instil presented posters highlighting pre-clinical data from the CoStAR platform demonstrating how CoStAR expression led to marked enhancement of effector function and proliferation of T cells and TILs in the studies. Further details can be found in the poster publications available on the publication section of the Company’s website.

Developing Novel CoStAR Constructs: Instil is developing novel, proprietary families of CoStAR constructs against new targets for solid tumors beyond the initial indications targeted for ITIL-306. Instil expects to select the next CoStAR candidate for IND-enabling studies in 2022.

Expansion of UK Manufacturing Facility Complete: Regulatory approval of the IMPACT manufacturing facility in Manchester, UK has been granted and clinical product manufacturing is ongoing. The opening of the IMPACT facility expands Instil’s clinical manufacturing capacity in Manchester, UK.

Validation of Tarzana Clinical Manufacturing Facility Ongoing: Validation of Instil’s clinical manufacturing facility in Tarzana, California is ongoing, with availability for clinical manufacturing expected in the first half of 2022. Upon readiness of the Tarzana clinical manufacturing facility, Instil expects to achieve manufacturing capacity of up to approximately 500 patient doses per year.
Fourth Quarter Full Year 2021 Financial and Operating Results:

As of December 31, 2021, we had $37.6 million in cash and cash equivalents and $416.5 million in marketable securities, compared to $241.7 million in cash and cash equivalents and no investments in marketable securities as of December 31, 2020. The Company expects that its cash, cash equivalents and marketable securities as of December 31, 2021 will enable it to fund its operating plan into 2023.

Research and development expenses were $42.6 million and $107.3 million for the fourth quarter and full year ended December 31, 2021, compared to $10.2 million and $19.4 million for the fourth quarter and full year ended December 31, 2020, respectively.

General and administrative expenses were $11.2 million and $48.3 million for the fourth quarter and full year ended December 31, 2021, compared to $7.2 million and $14.4 million for the fourth quarter and full year ended December 31, 2020, respectively

On March 7, 2022 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 full year ended December 31, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 7, 2022, View Source [SID1234609600]).

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"We are excited by the momentum and engagement around our ongoing Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy, as well as our DRAGON Phase 1 trial of SRK-181 in solid tumors. These programs have significant potential to address unmet patient need in a differentiated way, as well as demonstrate the tremendous potential of Scholar Rock’s platform, which focuses on inhibiting the activation of latent growth factors," said Nagesh Mahanthappa, Interim Chief Executive Officer of Scholar Rock. "While we progress these clinical programs, we also continue to build upon our world-leading expertise in selectively targeting the activation mechanisms of the TGFβ-superfamily growth factors and advance our preclinical pipeline focused on diseases such as fibrosis and regulation of iron metabolism. We look forward to providing more details about these programs at scientific meetings and through publications in the coming months."

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).

Conducting SAPPHIRE Phase 3 Clinical Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 SMA. The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab as add-on therapy for patients on either nusinersen or risdiplam. The study design plans for approximately 156 patients aged 2-12 years old with non-ambulatory Type 2 and 3 SMA to be enrolled in the main efficacy population. Patients will be randomized 1:1:1 to receive for 12 months either apitegromab 20 mg/kg, apitegromab 10 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks in addition to background SMN treatment.
Data from TOPAZ Phase 2 Open Label Extension Trial Expected to be Presented by mid-2022. As of February 28, 2022, 55 of 57 patients remain enrolled in the long-term extension trial of apitegromab in Type 2 and Type 3 SMA.
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming primary resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Advancing Part B of the DRAGON Phase 1 POC Trial for SRK-181. Based on the safety and pharmacokinetic data from Part A of the DRAGON Phase 1 trial, Scholar Rock is conducting the Part B dose expansion portion of the trial, in which SRK-181 is dosed at 1500 mg every three weeks (Q3W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q3W, or 1000 mg every two weeks (Q2W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q2W. Part B consists of multiple proof-of-concept cohorts focused upon evaluating the ability of SRK-181 to overcome primary resistance to anti-PD-(L)1 therapy. Each cohort will enroll up to 40 patients with various solid tumors, including urothelial carcinoma (UC), cutaneous melanoma (MEL), non-small cell lung cancer (NSCLC), clear cell renal cell carcinoma (ccRCC), and other solid tumors. The biomarker strategy in part B of DRAGON will explore early signs of SRK-181 activity, including target engagement and pathway modulation. This will include measuring effects on both circulating and tumor immune contexture, such as CD8+ T cell infiltration and reductions in myeloid-derived suppressor cell (MDSC) populations as well as analysis of TGFβ-related pathway signaling. Early efficacy and safety data are anticipated in 2022.
Full Year 2021 Financial Results

For the full year ended December 31, 2021, net loss was $131.8 million or $3.59 per share compared to a net loss of $86.5.0 million or $2.81 per share for the year ended December 31, 2020.

Revenue was $18.8 million for the year ended December 31, 2021 compared to $15.4 million for the year ended December 31, 2020. Revenue was related to the Gilead fibrosis-focused research collaboration, which concluded in December 2021.
Research and development expense was $108.5 million for the year ended December 31, 2021 compared to $74.1 million for the year ended December 31, 2020. The increase year-over-year was primarily attributable to planned spend associated with apitegromab development, including costs associated with clinical drug supply manufacturing and costs associated with our SAPPHIRE trial, as well as higher personnel-related costs.
General and administrative expense was $40.3 million for the year ended December 31, 2021 compared to $28.2 million for the year ended December 31, 2020. The increase year-over-year was primarily attributable to professional services and higher personnel-related costs, including filling key positions essential to progressing research, development and pre-commercial activities.
As of December 31, 2021, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $253.0 million, which is expected to fund the Company’s operations into mid-2023. "Continued operational excellence is an important priority for us in 2022 as we advance several programs across the continuum of drug development. As we continue to progress our SMA program with our Phase 3 SAPPHIRE trial, we are also encouraged that so many of the patients who participated in the TOPAZ study are still opting to receive apitegromab. We look forward to obtaining further insights into longer-term treatment outcomes and sharing that data in the coming months. In our immuno-oncology program, the DRAGON study has entered Part B and could produce meaningful data later this year and our research colleagues are pushing forward on several fronts as we continue to generate exciting programs from our scientific platform," said Ted Myles, COO and CFO of Scholar Rock.