On August 11, 2020 Context Therapeutics, a clinical-stage biopharmaceutical company dedicated to advancing medicines for hormone driven cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for onapristone extended release (ONA-XR), for the treatment of patients with progesterone receptor positive (PR+) ovarian cancer (Press release, Context Therapeutics, AUG 11, 2020, View Source [SID1234563433]).

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"We are very encouraged by the FDA’s decision to grant us Fast Track Designation for our lead program ONA-XR, which has shown very encouraging activity and tolerability in our ongoing Phase 2 study in advanced PR+ ovarian cancers. We believe this recognition underscores the high unmet medical need for a treatment for patients with PR+ ovarian cancer," said Martin Lehr, Chief Executive Officer of ContextTherapeutics. "With this designation in hand, we plan to be able to quickly advance through the administrative steps of ONA-XR’s development and bring forth a therapy for these patients as soon as possible."

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill unmet medical needs. A drug granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if certain criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a New Drug Application (NDA).

About Onapristone Extended Release
ONA-XR (onapristone extended release) is a potent and specific antagonist of the progesterone receptor that is orally administered. Currently, there are no approved therapies that selectively target PR+ cancers. Preliminary preclinical and clinical data suggest that ONA-XR has anticancer activity by inhibiting progesterone receptor binding to chromatin, downregulating cancer stem cell mobilization and blocking immune evasion. ONA-XR is currently the subject of an ongoing Phase 2 clinical trial in progesterone receptor positive ovarian cancer. Additional Phase 2 clinical trials in breast and endometrial cancers will initiate in 2020. ONA-XR is an investigational drug that has not been approved for marketing by any regulatory authority.

On August 11, 2020 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported its financial results for the second quarter 2020 and provided a corporate update (Press release, Gossamer Bio, AUG 11, 2020, View Source [SID1234563432]).

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"I hold tremendous pride in the progress made by the Gossamer team to date," said Sheila Gujrathi, M.D., Co-Founder and Chief Executive Officer of Gossamer. "We look forward to the continued advancement of our product candidates, and before year end, we are excited for the achievement of milestones across all four of our clinical programs."

Clinical-Stage Product Candidate Updates

GB001: Oral DP2 Antagonist for Eosinophilic Asthma and Chronic Rhinosinusitis (CRS)

Phase 2b LEDA study in patients with moderate-to-severe eosinophilic asthma is on track to report topline data in the second half 2020.
Phase 2 TITAN study in patients with chronic rhinosinusitis, both with and without nasal polyps, is also on track to report topline data in the second half 2020.
GB002: Inhaled PDGFR Inhibitor for Pulmonary Arterial Hypertension (PAH)

GB002 is currently being evaluated in an ongoing Phase 1b study in PAH. Given COVID-19 related delays in study enrollment, Gossamer anticipates reporting initial results from this study in the second half of this year.
Subject to developments in the ongoing COVID-19 pandemic, Gossamer plans to commence TORREY, a Phase 2 study in functional class II and III PAH patients in the second half of 2020. The primary endpoint for this 24-week study will be change in pulmonary vascular resistance (PVR) from baseline. The key secondary endpoint will be change from baseline in 6-minute walk distance at week 24.
GB004: Oral HIF-1α Stabilizer for Inflammatory Bowel Disease

Gossamer to present data from its completed Phase 1b study in patients with active mild-to-moderate ulcerative colitis (UC) at UEG Week Virtual 2020, which will take place October 11-13. Gossamer previously announced topline data from its completed Phase 1b study on May 12, 2020.
Subject to developments in the ongoing COVID-19 pandemic, Gossamer plans to initiate SHIFT-UC, a 12-week Phase 2 study of GB004 in patients with mild-to-moderate UC in the second half of 2020.
GB1275: Oral CD11b Modulator for Oncology Indications

Further Phase 1 data from KEYNOTE-A36, a Phase 1/2 study to evaluate GB1275 as a monotherapy and in combination with either KEYTRUDA (pembrolizumab) or chemotherapy in patients with selected solid tumors, are expected to be announced in the fourth quarter of 2020.
Financial Results for the Quarter Ended June 30, 2020

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of June 30, 2020, were $600.4 million. The Company expects the combination of current cash, cash equivalents and marketable securities, and access to our debt facility will be sufficient to fund its operating and capital expenditures into 2024.
Research and Development (R&D) Expenses: For the quarter ended June 30, 2020, R&D expenses were $38.7 million, compared to R&D expenses of $35.7 million for the same period in 2019.
In Process Research and Development (IPR&D) Expenses: For the quarter ended June 30, 2020, IPR&D expenses were $15.0 million, compared to $1.0 million for the same period in 2019. This increase reflects a $15.0 million upfront payment to Aerpio Pharmaceuticals, Inc. for the amendment to the in-license of GB004.
General and Administrative (G&A) Expenses: For the quarter ended June 30, 2020, G&A expenses were $11.7 million, compared to $9.7 million for the same period in 2019.
Net Loss: Net loss for the quarter ended June 30, 2020, was $66.9 million, or $1.00 per share, compared to a net loss of $44.5 million, or $0.74 per share, for the same period in 2019.

On August 11, 2020 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported preliminary, unaudited revenue and net product sales for the second quarter of 2020 (Press release, Supernus, AUG 11, 2020, View Source [SID1234563431]).

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Preliminary second quarter 2020 revenue consisted of net product sales of $124.0 million and royalty revenue of $2.7 million. Preliminary second quarter 2020 net product sales of $124.0 million increased 21% compared to the same period in 2019 due to higher net product sales of Trokendi XR and Oxtellar XR and the addition of $10.6 million of net product sales from the acquisition of the CNS portfolio of US WorldMeds, which closed on June 9, 2020.

The second quarter 2020 revenue results included herein are preliminary and are therefore subject to change.

Conference Call Details

The Company expects to report full second quarter 2020 results after 5:00 p.m. ET on Tuesday, August 18, 2020. The Company will hold a conference call hosted by Jack Khattar, President and Chief Executive Officer, and Greg Patrick, Senior Vice President and Chief Financial Officer, to discuss these results at 9:00 a.m. Eastern Time, on Wednesday, August 19, 2020. Please refer to the information below for conference call dial-in information and webcast registration. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference dial-in: (877) 288-1043
International dial-in: (970) 315-0267
Conference ID: 5175177
Conference Call Name: Supernus Pharmaceuticals Second Quarter 2020 Earnings Conference Call
Following the live call, a replay will be available on the Company’s website, www.supernus.com, under "Investor Relations".

On August 11, 2020 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53, reported financial results for the three and six months ended June 30, 2020 and provided a business update (Press release, Aprea, AUG 11, 2020, View Source [SID1234563430]).

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"The full enrollment in June of our Phase 3 clinical trial evaluating eprenetapopt (APR-246) with azacitidine for the treatment of front-line TP53 mutant myelodysplastic syndromes (MDS) was a major milestone for Aprea and we look forward to top-line data by year-end 2020," said Christian S. Schade, President and Chief Executive Officer of Aprea. "In addition, we continue to advance the clinical development of eprenetapopt in different clinical settings and have recently enrolled the first patient in our solid tumor clinical trial program."

Business Operations Update:

The Company is conducting, supporting and planning multiple clinical trials of eprenetapopt (APR-246):

Pivotal Phase 3 MDS Trial—During the second quarter of 2020, the Company completed the full enrollment of 154 patients in its pivotal Phase 3 randomized, controlled trial evaluating eprenetapopt with azacitidine as frontline therapy in HMA-naïve TP53 mutant myelodysplastic syndromes (MDS) patients with a primary endpoint of complete remission (CR) rate. The Company remains confident it will have top-line data available by year-end 2020.

Phase 2 MDS/AML Post-Transplant Trial—The Company is currently enrolling its single-arm, open-label Phase 2 trial evaluating eprenetapopt with azacitidine as post-transplant maintenance therapy in TP53 mutant MDS and acute myeloid leukemia (AML) patients who have received an allogeneic stem cell transplant. Though the Company had initially observed a decrease in both patient screening and patient enrollment as a result of the COVID-19 pandemic, patient screening activity has returned to expected levels and the trial has currently enrolled 24 out of 31 patients with a number of additional patients in screening. The Company anticipates completing enrollment of this trial in the third quarter of 2020.

Phase 1 AML Trial—The Company is currently enrolling its Phase 1 trial evaluating the safety, tolerability, and preliminary efficacy of eprenetapopt therapy in TP53 mutant AML patients. The lead-in portion of the trial evaluated the tolerability of eprenetapopt with venetoclax, with or without azacitidine, and no dose-limiting toxicities were observed in patients receiving either regimen. Based on these results, the Company has expanded the trial to treat approximately 30 additional frontline TP53 mutant AML patients with the combination of eprenetapopt, venetoclax and azacitidine. The Company also plans to activate a separate cohort in the trial to evaluate the combination of eprenetapopt with azacitidine in approximately 30 frontline TP53 mutant AML patients.

Phase 1 NHL Trial—To further assess eprenetapopt in hematological malignancies, the Company has designed and plans to conduct a Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) and mantle cell lymphoma (MCL) assessing eprenetapopt with venetoclax and rituximab, and eprenetapopt with ibrutinib. The Company is targeting the first patient to be enrolled in the second half of 2020.

Phase 1/2 Solid Tumor Trial—Based on in vivo data suggesting synergistic activity between eprenetapopt and immuno-therapy agents including anti-PD-1 antibody, the Company has designed and plans to conduct Phase 1/2 clinical trials in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy. The Company enrolled its first patient in August 2020.

APR-548 — The Company’s second product candidate, APR-548, is a pre-clinical, next-generation p53 reactivator with the potential for oral administration. APR-548 exhibits high oral bioavailability in preclinical testing and is being developed in an oral dosage form. The Company completed Investigational New Drug, or IND, enabling preclinical studies of APR-548 and filed an IND with the FDA. However, based on feedback from the FDA, the Company will not be able to initiate human clinical trials of APR-548 until it is able to provide additional information necessary to address questions raised by the FDA.
Second Quarter Financial Results

Cash and cash equivalents: As of June 30, 2020, the Company had $112.9 million of cash and cash equivalents compared to $130.1 million of cash and cash equivalents as of December 31, 2019. The Company expects cash burn for the full year 2020 to be between $35.0 million $40.0 million. The Company believes its cash and cash equivalents as of June 30, 2020 will be sufficient to meet its current projected operating requirements into 2023.

Research and Development (R&D) expenses: R&D expenses were $10.7 million for the quarter ended June 30, 2020, compared to $4.3 million for the comparable period in 2019. The increase in R&D expenses was primarily related to the advancement of the Company’s lead product candidate, eprenetapopt. In Q1 2019 the Company commenced a pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for frontline treatment of TP53 mutant MDS which completed enrollment in Q2 2020 and is supported by two ongoing Phase 1b/2 investigator initiated trials, one in the U.S. and one in France, testing eprenetapopt with azacitidine as frontline treatment in TP53 mutant MDS and AML patients. In addition, in Q1 2020, the Company continued enrolling patients in a Phase 2 post-transplant MDS/AML clinical trial began enrolling patients in a Phase 1 clinical trial in frontline and relapsed/refractory TP53 mutant AML assessing eprenetapopt with venetoclax with or without azacitidine.

General and Administrative (G&A) expenses: G&A expenses were $3.8 million for the quarter ended June 30, 2020, compared to $1.7 million for the comparable period in 2019. The increase in G&A expenses was primarily due to increased insurance expense, non-cash stock-based compensation, personnel related costs and commercial development expense.

Net loss: Net loss was $16.4 million, or $0.78 per share for the quarter ended June 30, 2020, compared to a net loss of $5.3 million, or $4.45 per share for the quarter ended June 30, 2019. The Company had 21,186,827 shares of common stock outstanding as of June 30, 2020.

On August 11, 2020 Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, reported second-quarter 2020 financial results (Press release, Pliant Therapeutics, AUG 11, 2020, View Source [SID1234563429]).

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"2020 has been a productive year as we’ve made progress on multiple fronts, including the initiation of a 12-week Phase 2a clinical trial evaluating PLN-74809 in patients with idiopathic pulmonary fibrosis, as well as dosing initial cohorts in our first-in-human trial of our Novartis partnered program, PLN-1474, for the potential treatment of liver fibrosis," said Bernard Coulie, M.D., Ph.D., chief executive officer and president of Pliant Therapeutics. "Bolstered by a strong cash position from our Series C financing in February and our IPO in June, we continue to advance our robust pipeline. We’re now focused on executing our Phase 2a program for PLN-74809 and look forward to sharing further updates on all of our programs."

Recent Highlights

Completed an initial public offering (IPO) and concurrent private placement with Novartis. The IPO priced at $16.00 per share, generating gross proceeds of $165.6 million including shares sold to the underwriters pursuant to the full exercise of their overallotment option before deducting underwriting discounts, commissions and other offering expenses payable by Pliant. The company also received $10.0 million in additional proceeds from a concurrent private placement with Novartis Institutes for BioMedical Research, Inc. at the public offering price of $16.00 per share. The Company’s common stock commenced trading on the Nasdaq Global Select Market under the ticker symbol "PLRX" on June 3rd, 2020.
Phase 2a 12-week trials of PLN-74809 in idiopathic pulmonary fibrosis (IPF) and primary sclerosing cholangitis (PSC) have resumed enrollment. Through close coordination with over 100 global trial sites, Pliant has continued to conduct site initiation activities throughout the COVID-19 pandemic. The Company is offering a hybrid approach to clinical trial participation with home-health solutions designed to minimize the requirements for visits to healthcare facilities, as well as a campaign to raise awareness of the IPF and PSC programs across patient communities and patient advocacy groups. Phase 2a clinical trial sites are currently enrolling patients in both the IPF and PSC trials.
Opened Investigational New Drug (IND) Application for PLN-74809 for treatment of acute respiratory distress syndrome (ARDS) associated with COVID-19. Pliant submitted the COVID-19 ARDS IND in June and the FDA has since issued a "safe to proceed" letter. The Company plans to initiate a Phase 2 trial evaluating safety, tolerability and pharmacokinetics (PK) of PLN-74809, as well as exploratory clinical outcome measures in patients hospitalized with severe and critical COVID-19 in the second half of 2020. This represents the fourth IND that Pliant has successfully opened since operations began in 2016.
Completed dosing of initial cohorts of a Phase 1 trial of PLN-1474. The Phase 1 trial is designed to evaluate safety and tolerability, as well as PK of PLN-1474 in approximately 100 healthy volunteers across a dose range compared to placebo. The Phase 1 trial site has experienced delays due to COVID-19; however, it is now open and expected to continue dosing the remaining cohorts with topline data expected in the first quarter of 2021, subject to further impact of the COVID-19 pandemic. PLN-1474 is partnered with Novartis.
COVID-19 Preparedness

Pliant has worked proactively to establish policies and procedures to enable the Company to operate safely and productively during the pandemic. The Company has experienced delays in clinical trial operations which will impact the expected timing of data readouts, but is working closely with clinical sites to continue site initiation activities in compliance with study protocols while observing government and institutional guidelines. The clinical site conducting Pliant’s Phase 2a PET trial of PLN-74809 in IPF remains closed to clinical research, but expects to resume trial activities in the third quarter. Pliant has made significant progress in opening trial sites for its Phase 2a 12-week IPF and PSC trials by offering a hybrid approach to clinical trial participation with home-health solutions designed to minimize the requirements for visits to healthcare facilities, as well as a campaign to raise awareness of the IPF and PSC programs across patient communities and patient advocacy groups. The Company intends to provide more specific guidance regarding clinical trial progress and the timing of data readouts as the impacts of the pandemic are better understood.

Second-Quarter 2020 Financial Results

Related party revenue was $3.6 million as compared to nil for the prior-year period.
Research and development expenses were $17.5 million, as compared to $13.1 million for the prior-year period. The increase was primarily related to higher external research and development expenses associated with the advancement of several programs and ongoing Phase 2 clinical trials.
General and administrative expenses were $3.0 million, as compared to $2.6 million for the same period in 2019. The increase was due to higher personnel-related and professional services expenses.
Net loss of $17.0 million as compared to a net loss of $15.5 million for the prior-year period.
As of June 30, 2020, Pliant had cash, cash equivalents and short-term investments of $312.5 million, compared to $102.8 million as of December 31, 2019. Pliant believes it has sufficient funds to meet its operating and capital requirements into 2023.