On February 17, 2020 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the fourth quarter and full year ended December 31, 2019, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets, for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, FEB 27, 2020, View Source [SID1234554906]).

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"We are entering 2020 with a clear plan to drive shareholder value for Rigel," said Raul Rodriguez, Rigel’s president and CEO. "We see substantial opportunities for TAVALISSE to meet patient needs in the growing adult chronic ITP market, particularly as an early line therapy. Utilization in these less refractory patients continues to grow with the support of ongoing physician education and data generation. To expand the range of TAVALISSE indications, we are conducting a Phase 3 trial in warm AIHA and expect to complete patient enrollment midyear. In addition, we are extremely excited about the potential of our early stage candidates and are currently exploring partnership opportunities that would enable Rigel to realize near-term value while also participating meaningfully in the upside of these programs."

Business Update

At the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in December 2019, the company presented post-hoc data analysis from its Phase 3 clinical program of TAVALISSE in adult patients with chronic ITP. In this analysis, 32 patients received fostamatinib as a second-line therapy, of which, 78% (25/32) achieved ≥1 platelet count of ≥50,000/µL (without rescue therapy). Adverse events were manageable and consistent with those previously reported with fostamatinib. These data highlight the potential benefit of using TAVALISSE in earlier lines of therapy.

In February 2020, Rigel received a $20.0 million payment from its collaborative partner Grifols, S.A. (Grifols). The payment is comprised of $17.5 million for the European Commission’s approval of the marketing authorization application for fostamatinib for the treatment of chronic immune thrombocytopenia in adult patients who are refractory to other treatments and a $2.5 million creditable advance royalty payment based on the terms of the collaboration agreement. Fostamatinib will be marketed in Europe under the brand name TAVLESSE (fostamatinib). Grifols is planning to launch the product in the second quarter of 2020.

Enrollment is ongoing in Rigel’s Phase 3 pivotal trial in warm AIHA, FORWARD (Fostamatinib Research in Warm Antibody AIHA Disease). A total of 34 patients have been randomized to date. The trial remains on track to complete enrollment in mid-2020.

In February 2020, Rigel’s partner Kissei Pharmaceuticals Co., Ltd. (Kissei) was granted orphan drug designation from the Japanese Ministry of Health, Labour and Welfare for R788 (fostamatinib) in chronic idiopathic thrombocytopenic purpura.

Financial Update

For the fourth quarter of 2019, Rigel reported a net loss of $17.2 million, or $0.10 per share, compared to net income of $3.2 million, or $0.02 per share, in the same period of 2018.

In the fourth quarter of 2019, total revenues were $15.4 million, consisting of $13.8 million in net product sales and $1.6 million in contract revenues from collaborations. Net product sales of $13.8 million increased by 90% compared to $7.3 million in the fourth quarter of 2018. This increase reflects the expanding patient and prescriber base for TAVALISSE and the growing persistency rate for refills at month 4, which is approximately 54%.

Contract revenues from collaborations of $1.6 million for the fourth quarter ended December 31, 2019 consists of a $1.5 million fee earned pursuant to an amendment of the license and collaboration agreement with Aclaris Therapeutics, Inc. (Aclaris) in October 2019, as well as deferred revenue from Rigel’s collaboration with Grifols related to the performance of certain research and development services.

Rigel reported total costs and expenses of $32.7 million in the fourth quarter of 2019, compared to $35.3 million for the same period in 2018. The decrease in costs and expenses was primarily due to decreases in personnel-related expenses and various third-party costs.

For the full year ended December 31, 2019, Rigel reported a net loss of $66.9 million, or $0.40 per share, compared to a net loss of $70.5 million, or $0.44 per share, for the same period of 2018.

Rigel reported total revenues of $59.3 million for the year ended December 31, 2019, consisting of $43.8 million in net product sales and $15.5 million in revenues related to Rigel’s collaboration agreements with Grifols, Kissei, Aclaris, and Impact Biomedicines, Inc.

Total costs and expenses for the year ended December 31, 2019, were $128.4 million, compared to $117.2 million, for the same period of 2018. The increase in total costs and expenses was primarily due to the increases in third party costs related to Rigel’s ongoing pivotal Phase 3 study in warm AIHA, personnel-related costs, on-going commercialization of TAVALISSE in adult chronic ITP, and research and development costs related to its Phase 1 study in RIP.

As of December 31, 2019, Rigel had cash, cash equivalents and short-term investments of $98.1 million, compared to $128.5 million as of December 31, 2018. Rigel previously announced that in September 2019, we entered into a $60.0 million term loan credit facility with MidCap Financial. At closing,

$10.0 million was funded to Rigel in an initial tranche. The facility also gives Rigel the ability to access an additional $50.0 million, of which $40.0 million is subject to the achievement of certain customary conditions.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time

Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call and accompanying slides will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP

In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA

Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 45,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients. Warm antibody AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature.

About R8351

The investigational candidate, R835, is an orally available, potent and selective inhibitor of IRAK1 and IRAK4 that has been shown preclinically to block inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response, and dysregulation of these pathways can lead to a variety of inflammatory pathological conditions. R835 treatment demonstrates amelioration of clinical symptoms in multiple rodent models of inflammatory disease including psoriasis, arthritis, lupus, multiple sclerosis and gout. The safety and efficacy of R835 has not been established by the FDA or any healthcare authority.

About R5521

The investigational candidate, R552, is an orally available, potent and selective inhibitor of receptor-interacting protein kinase (RIP1). RIP1 is believed to play a critical role in necroptosis. Necroptosis is a form of regulated cell death where the rupturing of cells leads to the dispersion of their inner contents, which induces immune responses and enhances inflammation. In preclinical studies, R552 prevented joint and skin inflammation in a RIP1-mediated murine model of inflammation and tissue damage. The safety and efficacy of R552 has not been established by the FDA or any healthcare authority.

About TAVALISSE

Indication

TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information

Warnings and Precautions

·Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
·Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
·Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
·Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
·TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.

Drug Interactions

·Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
·It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
·Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
·Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.

Adverse Reactions

·Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
·Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

Please see www.TAVALISSE.com for full Prescribing Information.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

TAVALISSE is a trademark of Rigel Pharmaceuticals, Inc.

On February 27, 2020 Trovagene, Inc. (Nasdaq: TROV), a clinical-stage, Precision Cancer Medicine oncology therapeutics company developing drugs that target cell division (mitosis) for the treatment of various cancers including prostate, colorectal and leukemia, reported company highlights and financial results for the fourth quarter and full-year ended December 31, 2019 (Press release, Trovagene, FEB 27, 2020, View Source [SID1234554905]). The company is issuing this press release in lieu of conducting a conference calll.

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"We continue to advance the clinical development of onvansertib, our first-in-class, third-generation, oral and highly selective Polo-like Kinase 1 (PLK1) inhibitor, including very encouraging to-date results from our Phase 1b/2 trial in KRAS-mutated metastatic colorectal cancer (mCRC)," said Dr. Thomas Adams, Chief Executive Officer and Chairman of Trovagene. "Regardless of the specific KRAS mutation, we are seeing quantitative decreases in the mutational burden of this once considered ‘undruggable’ mutation and confirmation of the activity of onvansertib by radiographic scans showing tumor regression in all treated patients. Additionally, in our Phase 2 study in metastatic castration-resistant prostate cancer (mCRPC), onvansertib is demonstrating its ability to overcome resistance in patients treated first-line with androgen-receptor signaling inhibitor, Zytiga. Data presented earlier this month at ASCO (Free ASCO Whitepaper)-GU show both efficacy and durability of response to treatment when onvansertib is added to daily Zytiga therapy. Notably, several patients currently on treatment have had progression-free survival of greater than seven months. Enrollment is also on track in our Phase 2 study of onvansertib in patients with acute myeloid leukemia (AML). We continue to see efficacy in this difficult-to-treat, relapsed/refractory patient population, including in patients who have relapsed following initial treatment with venetoclax, a scenario with limited treatment options and a great need for new targeted therapeutics."

The Company has advanced its business in the fourth quarter of 2019, and to-date in 2020, with the following activities and milestone achievements:

Announced presentation of Phase 2 data demonstrating the ability of onvansertib to overcome Zytiga-resistance and provide clinical benefit for mCRPC patients
On February 13, 2020, we announced the presentation of positive data from our ongoing Phase 2 trial of onvansertib in combination with Zytiga for the treatment of patients with Zytiga-resistant mCRPC. The data demonstrates the efficacy of onvansertib in Zytiga-resistant mCRPC across known androgen receptor resistance mechanisms. Additionally, onvansertib-induced decreases in circulating tumor cells (CTCs) is a surrogate for efficacy and associated with greater progression-free survival in mCRPC patients.

Announced data demonstrating the effectiveness of onvansertib as a pan-KRAS inhibitor with confirmed tumor regression and clinical benefit achieved in KRAS-mutated mCRC patients
On January 27, 2020, we announced the presentation of data from our ongoing Phase 1b/2 trial of onvansertib in combination with FOLFIRI/Avastin in patients with KRAS-muted mCRC. All patients treated showed tumor regression by radiographic scan at 8 weeks and confirmation by further tumor shrinkage at 16 weeks, with clinical benefit achieved in 100% of patients. Tumor regression occurred across all major KRAS mutations associated with mCRC, an indication of onvansertib’s pan-KRAS inhibitory effect; three patients had greater than 25% tumor shrinkage. One patient is proceeding to curative surgery, which is considered to be unprecedented in this patient population, in which there has been only a 5% response rate to standard-of-care therapy.

Announced positive data presented in an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") conference demonstrating efficacy, durability of response and safety of onvansertib in completed Phase 1b trial in AML
On December 9, 2019, we announced the presentation of data demonstrating efficacy, durability of response and safety of onvansertib from the completed Phase 1b segment of the ongoing trial in AML, in an oral session at ASH (Free ASH Whitepaper). Efficacy was observed in patients treated at onvansertib doses ranging from 27 to 90 mg/m2, with a complete response (CR) and CR with incomplete count recovery (Cri) rate of 31% (5 out of 16) of patients. Treatment was well tolerated; adverse events related to onvansertib were primarily on-target hematological (based on mechanism of action) and were easily managed and reversible. Biomarker positive patients showed a higher response to treatment; 67% (4 out of 6) of patients had marked decreases in bone marrow blast cells vs only 18% (1 out of 11) biomarker negative patients.

Announced data showing the ability of onvanserib to rescue patients previously treated with, and resistant to, venetoclax in AML
On December 4, 2019, we announced data showing the ability of onvansertib to rescue venetoclax-resistant AML patients. Patients develop resistance to venetoclax in approximately 11 months following the start of treatment with no viable therapeutic options and a median survival of only 1.7 to 2.3 months and a poor prognosis. Onvansertib as a single agent inhibits tumor

growth in both venetoclax-resistant in-vitro and in-vivo AML models. The combination of onvansertib and venetoclax is synergistic, which supports the addition of onvansertib to venetoclax in venetoclax-resistant AML patients.

Announced positive response to treatment in Phase 2 trial of onvansertib in patients with mCRPC
On November 14, 2019, we announced data demonstrating positive response to treatment in patients enrolled in our Phase 2 trial of onvansertib in combination with Zytiga (abiraterone acetate) in patients with mCRPC. 72% of patients had decreases in PSA levels with the addition of onvansertib following 1 cycle of treatment. 60% of patients completing 3 months of treatment and evaluable for efficacy achieved the primary endpoint of disease control.

Announced data presented at ESMO (Free ESMO Whitepaper) providing rationale for a clinical trial of onvansertib in subset of patients with Highly-Aggressive Triple Negative Breast Cancer (TNBC)
On October 2, 2019, we announced the presentation of data in a poster at ESMO (Free ESMO Whitepaper) demonstrating significant tumor regression observed with onvansertib in combination with standard-of-care paclitaxel in models of p53-mutated TNBC. Onvansertib’s preclinical data provides rationale for conducting a clinical trial targeting the 80% of TNBC that harbors the p53 mutation. The combination has potential to address critical medical needs to provide targeted treatment options to overcome resistance to paclitaxel as a single agent therapy in TNBC.
Fourth Quarter 2019 Financial Results
Total operating expenses were approximately $4.4 million for the three months ended December 31, 2019, an increase of $0.2 million from $4.2 million for the same period in 2018. The increase in operating expenses is attributed to advancing the onvansertib clinical development programs.
Net cash used in operating activities in the fourth quarter of 2019 was $3.3 million, compared to $3.6 million in the fourth quarter of 2018. The quarter-over-quarter decrease of $0.3 million can be attributed primarily to a favorable change in assets and liabilities offset by increased expenses pertaining to the clinical development of onvansertib.
Research and development expenses increased by approximately $0.4 million to $2.9 million for the three months ended December 31, 2019 from $2.5 million for the same period in 2018. The overall increase in research and development expenses was primarily due to the increased outside service costs for advancing clinical studies related to the development of our drug candidate, onvansertib. We expect increases in research and development costs to continue as we advance the onvansertib clinical development programs in mCRC, mCRPC and AML.
Selling, general and administrative expenses decreased by approximately $0.2 million to $1.5 million for the three months ended December 31, 2019 from $1.7 million for the same period in 2018. The reduction is primarily due to a decrease in outside services and professional fees.
The weighted average basic and diluted shares of common stock outstanding used to calculate per share results for the three months ended December 31, 2019 was 8.3 million.
As of December 31, 2019, Trovagene had approximately $10.2 million of cash and cash equivalents.

On February 27, 2020 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and utilizing a product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported financial results for the fourth quarter and year ended December 31, 2019 (Press release, Tracon Pharmaceuticals, FEB 27, 2020, View Source [SID1234554904]).

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Recent Corporate Highlights

In December, TRACON entered into a collaborative partnership agreement with 3D Medicines Co., Ltd. (3D Medicines) and Jiangsu Alphamab Biopharmaceuticals Co., Ltd. (Alphamab), for the North American development of envafolimab, a PD-L1 single-domain antibody administered by subcutaneous injection without an adjuvant, in soft tissue sarcoma. We expect to meet with the FDA to discuss a potential pivotal trial design and apply for orphan drug designation in the second quarter. In addition, we anticipate beginning patient enrollment in the study during the second half of this year.

In December and January, TRACON sold shares through our ATM facility with JonesTrading for gross proceeds of $5.3 million that extends the Company’s cash runway into 2021.

"We are pleased to have entered into this recent promising collaboration with 3D Medicines and Alphamab for the development and commercialization of envafolimab, a potentially best in class PD-L1 single domain antibody that may offer a much needed new treatment option for sarcoma patients," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Envafolimab has now become our lead asset. We are focused on beginning a pivotal trial as quickly as possible so we can address the high unmet medical need that exists for sarcoma patients, and have the opportunity to become a commercial company in the U.S. in approximately three years with envafolimab."

Expected Upcoming Milestones

Meet with FDA to discuss potential pivotal trial design for envafolimab, followed by the submission of an application for orphan drug designation, in the first half of 2020.

Enroll the first patient in a potentially pivotal trial of envafolimab in sarcoma during the second half of 2020.

Top-line data from the fully enrolled randomized Phase 2 AVANTE trial of DE-122 in patients with wet AMD, including the primary endpoint of mean change in best corrected visual acuity at six months, are expected in the first half of 2020. In this trial, the combination of DE-122 with Lucentis is being compared with Lucentis single agent treatment.

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URL: www.traconpharma.com

Phase 2 proof-of-concept data from the fully enrolled Phase 1/2 clinical trial of TRC253 in metastatic castrate resistant prostate cancer are expected in the first half of 2020. These clinical data will trigger Janssen’s 90-day option to reacquire full rights to TRC253 for an opt-in payment of $45 million to TRACON and obligations to pay regulatory and commercialization milestones totaling up to $137.5 million upon the achievement of specified events, in addition to a low single-digit royalty on net sales. If Janssen does not opt in, TRACON can maintain ownership of TRC253 and would owe Janssen up to $45 million upon the achievement of specified events, in addition to a low single-digit royalty on net sales.

Top-line data from the Phase 1 dose escalation study of TJ004309, a CD73 antibody, as a single agent and in combination with Tecentriq, a marketed PD-L1 antibody being supplied by Roche, are expected in the second half of 2020. We are developing TJ004309 in collaboration with our corporate partner I-Mab Biopharma.

Nomination and IND filing of the initial bispecific antibody (BsAb) from the I-Mab pipeline is expected in the second half of 2020. The I-Mab BsAb pipeline includes PD-L1 x IL-7, PD-L1 x CD47, PD-L1 x CD73, PD-L1 x B7-H3, and PD-L1 x 4-1BB antibodies. In 2018, TRACON and I-Mab entered into a cost-sharing product development collaboration, whereby TRACON will be responsible for the regulatory and clinical development of up to five of the BsAbs in North America and Europe, with the majority of the development effort expected to occur in the U.S. TRACON will bear the costs of early phase clinical trials and I-Mab will share the costs for more advanced development stages and commercialization. TRACON will share North American rights of any selected BsAbs with I-Mab for each collaborative program and TRACON retains an opt-in right to in-license ex-greater China rights to each of the selected BsAbs from I-Mab.

Fourth Quarter 2019 Financial Results

Cash, cash equivalents and short-term investments were $16.4 million at December 31, 2019, compared to $39.1 million at December 31, 2018. We expect our current cash, cash equivalents and short-term investments to fund operations into 2021.

Research and development expenses for the fourth quarter of 2019 were $1.9 million, compared to $5.9 million for the fourth quarter of 2018. The decrease was primarily attributable to lower manufacturing expenses and clinical trial expenses due to the discontinuation of the TRC105 program.

General and administrative expenses for the fourth quarter of 2019 were $1.9 million, compared to $1.8 million for the fourth quarter of 2018.

Net loss for the fourth quarter of 2019 was $3.9 million, compared to $7.8 million for the fourth quarter of 2018.

Investor Conference Call

The Company will hold a conference call today at 4:30 p.m. EDT / 1:30 p.m. PDT to provide an update on corporate activities and to discuss the financial results of its fourth quarter and full year of 2019. The dial-in numbers are (855) 779‑9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 3228345. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab is a novel, single-domain antibody against PD-L1 that is administered by subcutaneous injection without the need for an adjuvant. Envafolimab is currently dosing in Phase 1 trials in the U.S. and Japan and is being studied in China in a Phase 2 registration trial as a single agent in MSI-H tumor patients, and in combination with gemcitabine and oxaliplatin in a Phase 3 registration trial in biliary tract cancer. Subject to positive data from the MSI-H registrational trial, 3D Medicines plans to file a BLA in China for envafolimab in 2020 based on overall response rate in MSI-H patients. The filing would be based on the principle that the response rate required for approval in China is similar to the response rate for Keytruda and Opdivo in MSI-H patients from separate clinical trials per the product package inserts.

About DE-122 (carotuximab)

DE-122, an ophthalmic formulation of carotuximab, is active in preclinical choroidal neovascularization (CNV) models and designed to enhance the effect of approved VEGF inhibitors used to treat wet AMD. DE-122 is being investigated in the Phase 2 randomized AVANTE trial assessing the efficacy and safety of intravitreal injections in combination with Lucentis (ranibizumab) compared to Lucentis monotherapy in patients with wet AMD.

About TRC253

TRC253 is a novel, orally bioavailable small molecule drug that is a potent, high affinity competitive inhibitor of the androgen receptor (AR) and AR mutations, including the F877L mutation. The AR F877L mutation results in an alteration in the AR ligand binding domain that confers resistance to therapies for prostate cancer. Therapies targeting the AR have demonstrated clinical efficacy by extending time to disease progression, and in some cases, the survival of patients with metastatic castration-resistant prostate cancer. However, resistance to these agents is often observed and several molecular mechanisms of resistance have been identified, including gene amplification, overexpression, alternative splicing, and point mutation of the AR. TRC253 is currently being studied in a Phase 1/2 clinical trial in prostate cancer.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in a Phase 1 trial to assess safety and preliminary

efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.

On February 27, 2020 Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, provided a corporate update and reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Acceleron Pharma, FEB 27, 2020, View Source [SID1234554903]).

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"2019 was a pivotal year for Acceleron as we achieved the most important milestone to date in our 16-year history-our first U.S. FDA approval," said Habib Dable, President and Chief Executive Officer of Acceleron. "In November, REBLOZYL (luspatercept-aamt) became the first and only FDA approved erythroid maturation agent and is now indicated to treat anemia in adults with beta-thalassemia who require regular red blood cell transfusions. The approval of REBLOZYL marks a huge victory for patients with beta-thalassemia, who-up until now-have been waiting for a better way to treat their anemia. It is also a validation of Acceleron’s scientific platform leveraging our expertise in the TGF-beta superfamily of proteins and our unwavering commitment to develop and commercialize medicines to treat serious and rare diseases."

Continued Mr. Dable: "While our hematology program remains a top priority, we are equally excited by the progress we are making in pulmonary disease. We recently announced positive topline results from the PULSAR Phase 2 trial of sotatercept for the treatment of PAH. These data introduce the possibility that sotatercept could potentially change the way physicians currently treat patients with PAH. We look forward to presenting the detailed topline results at the American Thoracic Society 2020 International Conference in May and to our upcoming interactions with health authorities as we plan to globally develop and, if approved, commercialize sotatercept in PAH. We also expect to announce topline results from our Phase 2 neuromuscular program in Charcot-Marie-Tooth disease in March."

Development Program Highlights

Hematology

REBLOZYL (luspatercept-aamt): Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
REBLOZYL is the first and only U.S. FDA approved erythroid maturation agent designed to promote red blood cell production through a novel mechanism. Luspatercept-aamt is being developed to treat anemia in patients with beta-thalassemia, MDS, or MF. REBLOZYL is part of the global collaboration between Acceleron and Bristol-Myers Squibb.

In November 2019, Acceleron and partner Bristol-Myers Squibb announced the FDA approved REBLOZYL for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions. The companies are also seeking approval of luspatercept-aamt for the treatment of anemia in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who have ring sideroblasts and require red blood cell (RBC) transfusions. The FDA target action date for the MDS indication is April 4, 2020.

In January 2020, the New England Journal of Medicine published results from MEDALIST, the pivotal Phase 3 study evaluating the use of luspatercept-aamt to treat anemia in adult patients with very low- to intermediate-risk MDS who have ring sideroblasts and require RBC transfusions.

Six clinical abstracts of luspatercept-aamt were presented at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando in December 2019, including:

Long-term results from two pivotal Phase 3 trials, the MEDALIST and BELIEVE trials;

Initial results from a Phase 2 trial in patients with anemia associated with MF.

The Company and partner Bristol-Myers Squibb announced plans for a pivotal Phase 3 study, called INDEPENDENCE, of luspatercept-aamt in adult patients with anemia associated with MF who are being treated with JAK inhibitor therapy and require RBC transfusions. The study is expected to open in 2020.

Pulmonary Disease

Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept (RAP-011) reversed pulmonary vessel muscularization and improved indicators of right heart failure.

In January 2020, Acceleron reported that the PULSAR Phase 2 trial in patients with PAH met its primary and key secondary endpoints. Sotatercept was generally well tolerated in the trial and adverse events observed in the study were generally consistent with previously published data on sotatercept in other diseases.

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The PULSAR topline results are expected to be presented at the American Thoracic Society’s (ATS) 2020 International Conference in Philadelphia, Pennsylvania, on May 15 to 20, 2020.

Enrollment is ongoing in the SPECTRA trial in patients with PAH, with preliminary results expected in 2020.

Additional Pulmonary Updates

The Company entered into a collaboration and license agreement with Fulcrum Therapeutics to identify small molecules designed to modulate specific pathways associated with a targeted indication within the pulmonary disease space.

Neuromuscular Disease

ACE-083: Charcot-Marie-Tooth Disease (CMT)
ACE-083 is an investigational locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause focal muscle weakness. ACE-083 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta superfamily ligands involved in muscle formation.

Topline results from Part 2 of the Phase 2 trial in patients with CMT are expected in March 2020.

Corporate Highlights

In December, Jay T. Backstrom, M.D., M.P.H., formerly Chief Medical Officer of Celgene, joined Acceleron’s senior leadership team as Head of Research and Development.

Kemal Malik, M.B. B.S., was appointed to the Board of Directors effective January 1, 2020. Dr. Malik has served on the Board of Management of Bayer AG since 2014, with responsibility for Innovation across the Bayer group.

Financial Results

Cash Position – Cash, cash equivalents and investments as of December 31, 2019 were $453.8 million. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from REBLOZYL sales.

Revenue – Collaboration revenue for the year was $74.0 million. The revenue was all derived from the Company’s partnership with Bristol-Myers Squibb and is primarily related to expenses incurred by the Company in support of REBLOZYL, as well as one-time gross milestone payments totaling $60.0 million earned upon the FDA acceptance of the BLA and EMA validation of the MAA of REBLOZYL in June, and the FDA approval of REBLOZYL in November 2019.

Costs and Expenses – Total costs and expenses for the year were $210.4 million. This included R&D expenses of $154.0 million and G&A expenses of $56.4 million.

Net Loss – The Company’s net loss for the year ended December 31, 2019 was $124.9 million.

Conference Call and Webcast
The Company will host a webcast and conference call to discuss its fourth quarter and full year financial results for 2019 and provide an update on recent corporate activities on February 27, 2020, at 5:00 p.m. EST.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron Fourth Quarter and Full Year 2019 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On February 27, 2020 Immunomedics, Inc. (NASDAQ: IMMU) ("Immunomedics" or the "Company"), a leading biopharmaceutical company in the area of antibody-drug conjugates, reported financial results for the fourth quarter and full year 2019 (Press release, Immunomedics, FEB 27, 2020, View Source [SID1234554902]). Please refer to the Company’s Annual Report on Form 10-K for more details on the Company’s financial results.

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"We are pleased to have submitted our Biologics License Application (BLA) in late November – our top priority for 2019," said Dr. Behzad Aghazadeh, executive chairman of Immunomedics. "While we are working closely with the FDA to facilitate their review of our BLA, we continued to execute against key initiatives across our commercial, clinical, and manufacturing operations. Our commercial team has been actively preparing for launch readiness, and, as we wait for an FDA decision, we are well positioned to commercialize sacituzumab govitecan in metastatic triple-negative breast cancer (mTNBC) on or before the June 2, 2020 PDUFA goal date. In addition, our main goals for 2020 include maximizing the global commercial potential of sacituzumab govitecan, while accelerating its development as a single agent and in combination with checkpoint inhibitors across a number of clinical settings to meet our commitment to help transform the lives of people with hard-to-treat cancers worldwide."

2019 Highlights

Resubmitted BLA seeking accelerated approval for sacituzumab govitecan in mTNBC, which was accepted for filing by the FDA with a PDUFA target action date of June 2, 2020.
Completed enrollment of the Phase 3 confirmatory ASCENT study of sacituzumab govitecan in mTNBC.

Initiated the Phase 3 registrational TROPiCS-02 study of sacituzumab govitecan in hormone receptor-positive/human epidermal growth factor receptor 2-negative (HER2‒) metastatic breast cancer.

Continued to expand clinical collaborations to move sacituzumab govitecan into earlier lines of therapy in breast cancer, including the combination with Roche’s Tecentriq in frontline mTNBC and the German Breast Group’s Phase 3 study in the curative setting of HER2‒ breast cancer as a single-agent.

Corroborated the positive Phase 2 results of sacituzumab govitecan in metastatic urothelial cancer with an interim 29% overall response rate presented at ESMO (Free ESMO Whitepaper) 2019 Congress on 35 patients, while completing enrollment of the full cohort of 100 patients previously treated with platinum-based chemotherapies and immune checkpoint inhibitor.

Initiated the multi-cohort, Trop-2-enriched Phase 2 TROPiCS-03 study of sacituzumab govitecan for patients with metastatic non-small cell lung cancer (mNSCLC).

Closed the largest single-asset biologics license deal for Greater China to date, with $65 million upfront, $60 million to be received upon FDA approval, up to $710 million in additional development and sales milestone payments, and escalating royalties that begin in the mid-teens.
These accomplishments have positioned Immunomedics to have a transformative 2020. We entered the year with a robust balance sheet that will enable us to meet our goals.

The Company continued to bolster the leadership team, both at the board and the executive management level, with the nomination of Robert Azelby to the board, and the permanent appointment of Dr. Loretta Itri to the chief medical officer position.

The company has recently also broadened the development program for sacituzumab govitecan by expanding the TROPHY U-01 study to include a third cohort of checkpoint inhibitor-naïve patients to assess the combination with pembrolizumab. Furthermore, a new Phase 2 study in persistent or recurrent endometrial cancer was launched by Dr. Alessandro Santin, Professor of Obstetrics, Gynecology, and Reproductive Sciences at Yale University. Finally, to help alleviate the unmet need of mTNBC patients between now and potential approval of sacituzumab govitecan, the Company instituted its first Expanded Access Program in January 2020. Details of this program can be obtained from View Source

Anticipated Key 2020 Milestones

Besides the FDA action date in the second quarter and a potential commercial launch, we anticipate the following key milestones in 2020:

Topline data from Phase 3 confirmatory ASCENT study around mid-year
Topline data from full cohort of 100 patients with prior platinum-based and immune checkpoint inhibitor therapies from Phase 2 pivotal TROPHY U-01 study
Enrollment completion for the Phase 3 registrational TROPiCS-02 study
Enrollment completion for the 40-patient cohort of cisplatin-ineligible patients in TROPHY U-01
Evaluate preliminary results from signal-seeking Phase 2 TROPiCS-03 study in mNSCLC
Continue to expand combination development opportunities between sacituzumab govitecan and checkpoint inhibitors across indications, internally and through partnerships
Fourth Quarter and Full Year 2019 Financial Results

Total revenues were $0.3 million for both the quarter and the year ended December 31, 2019 due to service fee revenues earned related to the Janssen Promotion Agreement. The Company had no revenues in 2018 for the comparable quarter ended, and $0.9 million for the year ended December 31, 2018.

Total costs and expenses were $91.5 million for the quarter and $325.1 million for the year ended December 31, 2019, compared to $87.4 million for the comparable quarter and $235.4 million for the year ended December 31, 2018. The increases were primarily due to an increase in research and development expenses, mostly attributable to activities related to preparations for the approval and commercial launch of sacituzumab govitecan for patients with at least two prior lines of treatment for mTNBC in the United States, and CRL remediation costs, including outside manufacturers’ organizations services costs, and external consulting services to improve our manufacturing and regulatory functions.

The increases in research and development costs were partially offset by a decrease in general and administrative expenses, which was primarily due to decreased legal and advisory expenses resulted from reduced reliance on outside legal counsel, as well as a decrease in other and consulting services, partially offset by an increase in labor costs.

The Company had no non-cash warrant-related income or expense for the quarter and the year ended December 31, 2019, compared to a $0.2 million of non-cash gain for the comparable quarter and a $47.6 million warrant-related expense for the year ended December 31, 2018, due to changes in the fair value of then outstanding warrants. There were no warrants outstanding at December 31, 2019 and 2018.

Interest expense was $10.0 million for the quarter and $40.3 million for the year ended December 31, 2019, compared to $9.9 million for the comparable quarter and $40.4 million for the year ended December 31, 2018. The differences were due primarily to changes in the fair value of our debt balances as a result of the agreement with RPI Finance Trust.

Net loss attributable to stockholders was $99.6 million, or $0.50 per share, for the quarter ended December 31, 2019, compared to $93.5 million, or $0.50 per share, for the comparable quarter ended December 31, 2018. Net loss attributable to stockholders was $357.2 million, or $1.84 per share, for the year ended December 31, 2019, compared to $310.2 million, or $1.74 per share, for the year ended December 31, 2018.

As of December 31, 2019, the Company had $613.2 million in cash, cash equivalents, and marketable securities. The Company believes this amount is adequate to accelerate commercial launch readiness, pending FDA approval, of sacituzumab govitecan in the United States in mTNBC; continue to expand the clinical development programs for sacituzumab govitecan; invest in the broader clinical development of the ADC platform (including IMMU-130 and IMMU-140); continued scale up of manufacturing and manufacturing process improvements; and general working capital requirements.

Conference Call

The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Time to discuss fourth quarter and full year 2019 financial results and provide a corporate update. To access the conference call, please dial (877) 303-2523 or (253) 237-1755 using the Conference ID 4365097. The conference call will be webcast via the Investors page on the Company’s website at View Source Approximately two hours following the live event, a webcast replay of the conference call will be available on the Company’s website for approximately 30 days.