On May 4, 2021 Incyte (Nasdaq: INCY) reported 2021 first quarter financial results, and provides a status update on the Company’s development portfolio (Press release, Incyte, MAY 4, 2021, View Source [SID1234579097]).

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"In the first quarter, we continued to make significant progress in our strategy to drive growth and diversification. While Jakafi (ruxolitinib) net sales were affected by typical seasonal effects and softer patient demand growth due to the ongoing pandemic, we remain confident in our full-year outlook. We are already seeing a return of new patient starts to pre-COVID levels and are excited for the potential launch in steroid-refractory chronic graft-versus-host disease (GVHD) later this year. The launches of Monjuvi (tafasitamab) and Pemazyre (pemigatinib) continue to progress with good uptake by both academic and community physicians," stated Hervé Hoppenot, Chief Executive Officer, Incyte. "We expect an exciting year ahead for Incyte with the potential for multiple approvals, including ruxolitinib cream in atopic dermatitis, and several regulatory filings, notably parsaclisib in NHL and ruxolitinib cream in vitiligo. We are also initiating pivotal trials across key development programs for both tafasitamab and LIMBER this year."

Portfolio Update

MPNs and GVHD – key highlights

Ruxolitinib in GVHD: The supplemental New Drug Application (sNDA) seeking approval of ruxolitinib for the treatment of steroid-refractory chronic GVHD has been accepted for Priority Review by the U.S. Food and Drug Administration (FDA); the Prescription Drug User Fee Act (PDUFA) date is June 22, 2021. The application for approval was based on the successful randomized REACH3 trial comparing ruxolitinib with best available therapy (BAT).

LIMBER: Our Leadership In MPNs BEyond Ruxolitinib (LIMBER) development program continues to progress with once daily (QD) ruxolitinib in stability testing, and multiple ongoing and planned combination trials with ruxolitinib on track. Both monotherapy trials of INCB57643 (BET) and INCB00928 (ALK2) are ongoing, and combination trials of both agents with ruxolitinib in patients with myelofibrosis (MF) are expected to initiate later this year. Two Phase 3 trials of ruxolitinib in combination with parsaclisib as a first-line therapy for patients with MF (LIMBER-313) and as a therapy for MF patients with a suboptimal response to ruxolitinib monotherapy (LIMBER-304) are ongoing.

Indication and status

Once-a-day ruxolitinib
(JAK1/JAK2)

Myelofibrosis, polycythemia vera & GVHD: clinical pharmacology studies

ruxolitinib + parsaclisib
(JAK1/JAK2 + PI3Kδ)

Myelofibrosis: Phase 3 (first-line therapy) (LIMBER-313)

Myelofibrosis: Phase 3 (suboptimal responders to ruxolitinib) (LIMBER-304)

ruxolitinib + INCB57643
(JAK1/JAK2 + BET)

Myelofibrosis: Phase 2 in preparation

ruxolitinib + INCB00928
(JAK1/JAK2 + ALK2)

Myelofibrosis: Phase 2 in preparation

itacitinib
(JAK1)

Myelofibrosis: Phase 2 (low platelets)

ruxolitinib + CK08041
(JAK1/JAK2 + CB-Tregs)

Myelofibrosis: PoC in preparation

ruxolitinib
(JAK1/JAK2)

Steroid-refractory chronic GVHD2: sNDA under Priority Review

itacitinib
(JAK1)

Treatment-naïve chronic GVHD: Phase 3 (GRAVITAS-309)

Development collaboration with Cellenkos, Inc.
Clinical development of ruxolitinib in GVHD conducted in collaboration with Novartis
Other Hematology/Oncology – key highlights

Tafasitamab: Incyte and MorphoSys strategy to evaluate tafasitamab as a backbone therapy in multiple combinations for the treatment of various B-cell malignancies is underway. The Phase 3 inMIND trial, evaluating tafasitamab in combination with lenalidomide plus rituximab (R2) versus R2 in patients with follicular lymphoma and marginal zone lymphoma is ongoing. Preparations are underway for the Phase 3 frontMIND trial evaluating tafasitamab plus lenalidomide in addition to rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP) compared to R-CHOP alone as a first-line treatment for patients with newly diagnosed diffuse-large B-cell lymphoma (DLBCL). A proof of concept trial evaluating tafasitamab plus parsaclisib (topMIND) is expected to start later this year and a second proof of concept trial with tafasitamab plus lenalidomide in addition to plamotamab is expected to start later this year or early next.

Pemigatinib: In March, Pemazyre (pemigatinib) was approved by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients with unresectable biliary tract cancer (BTC) with a fibroblast growth factor receptor 2 (FGFR2) fusion gene, worsening after cancer chemotherapy. Additionally, the European Commission (EC) approved Pemazyre for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy. The upcoming launches in Europe and Japan along with the continued launch in the U.S., signifies the first internally discovered product to be globally commercialized by Incyte. The Phase 2 tumor agnostic study of pemigatinib is ongoing and continues to enroll well.

Retifanlimab: In March, the Marketing Authorization Application (MAA) seeking approval of retifanlimab in squamous cell carcinoma of the anal canal (SCAC) was validated by the European Medicines Agency (EMA). This follows the Biologics License Application (BLA) acceptance by the FDA for the same indication earlier this year.

Indication and status

pemigatinib
(FGFR1/2/3)

CCA: Phase 2 (FIGHT-202), Phase 3 (FIGHT-302)

8p11 MPN: Phase 2 (FIGHT-203)

Tumor agnostic: Phase 2 (FIGHT-207)

tafasitamab

(CD19)1

r/r DLBCL: Phase 2 (L-MIND); Phase 3 (B-MIND); MAA under review

1L DLBCL: Phase 1b (firstMIND); Phase 3 (frontMIND) in preparation

r/r FL and r/r MZL: Phase 3 (inMIND)

r/r B-cell malignancies: PoC (topMIND) with parsaclisib (PI3Kδ) in preparation

r/r B-cell malignancies: PoC with lenalidomide and plamotamab in preparation2

parsaclisib
(PI3Kδ)

r/r FL: Phase 2 (CITADEL-203)

r/r MZL: Phase 2 (CITADEL-204)

r/r MCL: Phase 2 (CITADEL-205)
r/r FL and r/r MZL: Phase 3 (CITADEL-302) in preparation
1L MCL: Phase 3 (CITADEL-310) in preparation

retifanlimab
(PD-1)3

SCAC: Phase 2 (POD1UM-202); Phase 3 (POD1UM-303); BLA and MAA under review

MSI-high endometrial cancer: Phase 2 (POD1UM-101, POD1UM-204)

Merkel cell carcinoma: Phase 2 (POD1UM-201)

NSCLC: Phase 3 (POD1UM-304)

CCA = cholangiocarcinoma; DLBCL = diffuse large B-cell lymphoma; SCAC = squamous cell anal carcinoma; FL = follicular lymphoma; MZL = marginal zone lymphoma; MCL = mantle cell lymphoma

Development of tafasitamab in collaboration with MorphoSys
Clinical collaboration with MorphoSys and Xencor, Inc. to investigate the combination of tafasitamab plus lenalidomide in combination with Xencor’s CD20xCD3 XmAb bispecific antibody, plamotamab.
retifanlimab licensed from MacroGenics
Inflammation and Autoimmunity (IAI) – key highlights

Dermatology

Ruxolitinib cream: The NDA seeking approval of ruxolitinib cream for the treatment of atopic dermatitis (AD) has been accepted for Priority Review by the FDA and the PDUFA date has been set for June 21, 2021. The application is supported by data from the Phase 3 TRuE-AD clinical trial program – pooled results presented last month at the American Academy of Dermatology (AAD) virtual conference provided additional context regarding the impact of ruxolitinib cream on itch relief as well as sleep disturbance and sleep impairment.

Also at AAD, Incyte presented 104-week data from the Phase 2 program evaluating ruxolitinib cream in patients with vitiligo. Results showed patients on ruxolitinib cream achieved continued efficacy through 104 weeks, with a longer duration of treatment being associated with greater levels of repigmentation. Incyte also presented additional follow-up data which demonstrated a potential for maintenance of repigmentation after discontinuation of therapy (following two years of treatment with ruxolitinib cream). The two Phase 3 trials in the TRuE-V program are ongoing with results expected in Q2’21.

INCB54707: In March, Incyte initiated a Phase 2 trial evaluating INCB54707 in vitiligo. A Phase 2 trial in hidradenitis suppurativa remains ongoing.

Indication and status

ruxolitinib cream
(JAK1/JAK2)

Atopic dermatitis: NDA under Priority Review

Vitiligo: Phase 3 (TRuE-V1, TRuE-V2; recruitment complete in both trials)

INCB54707
(JAK1)

Hidradenitis suppurativa: Phase 2b

Vitiligo: Phase 2

parsaclisib
(PI3Kδ)

Autoimmune hemolytic anemia: Phase 2

INCB00928
(ALK2)

Fibrodysplasia ossificans progressiva: Phase 2 in preparation

Discovery and early development – key highlights

INCB106385/INCA00186: At AACR (Free AACR Whitepaper), Incyte shared clinical and pre-clinical data from INCB106385, our novel A2A/A2B adenosine receptor antagonist, and INCA00186, our novel CD73 monoclonal antibody—both of which highlight our ongoing efforts targeting the adenosine pathway. Incyte also presented preclinical data for our triplet combination with retifanlimab, highlighting one of our strategies to overcome adenosine mediated PD-1 suppression.

Modality

Candidates

Small molecules

INCB01158 (ARG)1, INCB81776 (AXL/MER), epacadostat (IDO1), INCB86550 (PD-L1), INCB106385 (A2A/A2B)

Monoclonal antibodies2

INCAGN1876 (GITR), INCAGN2385 (LAG-3), INCAGN1949 (OX40), INCAGN2390 (TIM-3), INCA00186 (CD73)

Bispecific antibodies

MCLA-145 (PD-L1xCD137)3

INCB01158 development in collaboration with Calithera
Discovery collaboration with Agenus
MCLA-145 development in collaboration with Merus
Partnered – key highlights

Baricitinib: In April, Incyte and Lilly announced the FDA extended the review period for the sNDA for baricitinib for the treatment of moderate to severe atopic dermatitis by three months to allow time for additional data analyses. The PDUFA action date is now expected in early Q3.

In March, Incyte and Lilly announced positive results from BRAVE-AA2 evaluating the efficacy and safety of once-daily baricitinib 2-mg and 4-mg in adults with severe alopecia areata (AA), an autoimmune disorder that can cause unpredictable hair loss on the scalp, face and other areas of the body. In April, positive results from a second Phase 3 study, BRAVE-AA1, were announced, with data consistent with the findings from BRAVE-AA2. Lilly plans to submit an sNDA to the FDA for baricitinib in AA in the second half of 2021. There are currently no FDA-approved therapies for AA, highlighting the potential for baricitinib to address a significant unmet medical need.

Indication and status

baricitinib
(JAK1/JAK2)1

Atopic dermatitis: Phase 3 (BREEZE-AD); approved in EU and Japan; sNDA under review

Severe alopecia areata: Phase 3 (BRAVE-AA1, BRAVE-AA2)

Systemic lupus erythematosus: Phase 3 (BRAVE I, BRAVE II)

capmatinib
(MET)2

NSCLC (with MET exon 14 skipping mutations): Approved as Tabrecta in U.S. and Japan

Worldwide rights to baricitinib licensed to Lilly: approved as Olumiant in multiple territories globally for certain patients with moderate-to-severe rheumatoid arthritis; approved as Olumiant in EU and Japan for certain patients with atopic dermatitis
Worldwide rights to capmatinib licensed to Novartis
Potential therapies for patients with COVID-19

Ruxolitinib: In March, Incyte announced results from the Phase 3 DEVENT study assessing ruxolitinib plus standard of care (SoC) versus SoC in patients on mechanical ventilation with COVID-19 associated Acute Respiratory Disease Syndrome (ARDS). While results indicate a trend towards improvement in mortality in the overall study population, statistical significance was not reached in the primary endpoint (mortality due to any cause through Day 29). In the U.S. study population (91% of total study participants), however, there was a clinically and statistically significant improvement in mortality in each of the 5mg and 15mg ruxolitinib arms versus placebo.

Baricitinib: In April, Incyte and Lilly announced the primary endpoint in the Phase 3 COV-BARRIER study evaluating baricitinib in hospitalized COVID-19 patients – difference in the proportion of participants progressing to the first occurrence of non-invasive ventilation including high flow oxygen or invasive mechanical ventilation including extracorporeal membrane oxygenation (ECMO) or death by Day 28) – was not met. There was, however, a 38% reduction in mortality by Day 28 in patients treated with baricitinib in addition to SoC. Lilly will share these data with regulatory authorities in the U.S., European Union and other geographies to evaluate next steps.

2021 First Quarter Financial Results

The financial measures presented in this press release for the three months ended March 31, 2021 and 2020 have been prepared by the Company in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"), unless otherwise identified as a Non-GAAP financial measure. Management believes that Non-GAAP information is useful for investors, when considered in conjunction with Incyte’s GAAP disclosures. Management uses such information internally and externally for establishing budgets, operating goals and financial planning purposes. These metrics are also used to manage the Company’s business and monitor performance. The Company adjusts, where appropriate, for expenses in order to reflect the Company’s core operations. The Company believes these adjustments are useful to investors by providing an enhanced understanding of the financial performance of the Company’s core operations. The metrics have been adopted to align the Company with disclosures provided by industry peers.

Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used in conjunction with and to supplement Incyte’s operating results as reported under GAAP. Non-GAAP measures may be defined and calculated differently by other companies in our industry.

Financial Highlights

Product and Royalty Revenues Product and royalty revenues for the quarter ended March 31, 2021 increased 6% over the prior year comparative period as a result of increases in Jakafi net product revenues, the launch of Pemazyre and higher product royalty revenues from Jakavi and Olumiant. The year-over-year growth rate in Jakafi net product revenues was impacted by a decline in new patient starts due to the COVID-19 pandemic, and higher patient demand and channel inventory stocking in the prior year comparative period, due to concerns over potential COVID-19 related supply disruptions.

1. Non-GAAP cost of product revenues excludes the amortization of licensed intellectual property for Iclusig relating to the acquisition of the European business of ARIAD Pharmaceuticals, Inc. and the cost of stock-based compensation.
2. Non-GAAP research and development expenses exclude the cost of stock-based compensation.
3. Non-GAAP selling, general and administrative expenses exclude the cost of stock-based compensation and a legal reserve.
4. Non-GAAP change in fair value of acquisition-related contingent consideration is null.

Research and development expenses GAAP and Non-GAAP research and development expense for the quarter ended March 31, 2021 decreased 72% and 74%, respectively, compared to the same period in 2020, primarily due to upfront consideration of $805 million related to our collaborative agreement with MorphoSys incurred during the quarter ended March 31, 2020. Excluding the impact of upfront consideration and milestones, research and development expense for the quarter ended March 31, 2021 increased approximately 5% compared to the same period in 2020 reflecting costs to support the continued progression of our pipeline of programs.

Selling, general and administrative expenses GAAP and Non-GAAP selling, general and administrative expenses for the quarter ended March 31, 2021 increased 38% and 26%, respectively, compared to the same period in 2020, primarily due to expenses related to the establishment of our dermatology commercial organization and activities to support the potential launch of ruxolitinib cream for the treatment of atopic dermatitis, a $13 million reserve related to a settlement in principle in connection with the 2018 civil investigative demand from the U.S. Department of Justice, and the timing of certain expenses. Excluding the impact of the legal reserve, GAAP selling, general and administrative expenses for the quarter ended March 31, 2021 increased approximately 26% compared to the same period in 2020.

Other Financial Information

Operating income (loss) GAAP and Non-GAAP operating income (loss) for the quarter ended March 31, 2021 increased compared to the same period in 2020 primarily due to the decrease in upfront consideration and milestones related to our collaborative agreements and the growth in product and royalty revenues.

Cash, cash equivalents and marketable securities position As of March 31, 2021 and December 31, 2020, cash, cash equivalents and marketable securities totaled $2.0 billion and $1.8 billion, respectively.

2021 Financial Guidance

The Company has reaffirmed its full year 2021 financial guidance, as detailed below. Guidance does not include revenue from any potential new product launches. However, GAAP and Non-GAAP selling, general and administrative expense guidance for 2021 includes costs to support the potential launches of ruxolitinib cream as a treatment for atopic dermatitis in the U.S., pemigatinib as a treatment for cholangiocarcinoma in the EU and Japan, and tafasitamab as a treatment for DLBCL in the EU. The 2021 financial guidance does not include the impact of any potential future strategic transactions.

Unchanged

1. Adjusted to exclude the amortization of licensed intellectual property for Iclusig relating to the acquisition of the European business of ARIAD Pharmaceuticals, Inc. and the estimated cost of stock-based compensation.
2. Adjusted to exclude the estimated cost of stock-based compensation.
3. Adjusted to exclude the estimated cost of stock-based compensation and a legal reserve.

Conference Call and Webcast Information

Incyte will hold a conference call and webcast this morning at 8:00 a.m. ET. To access the conference call, please dial 877-407-3042 for domestic callers or 201-389-0864 for international callers. When prompted, provide the conference identification number, 13718346.

If you are unable to participate, a replay of the conference call will be available for 90 days. The replay dial-in number for the United States is 877-660-6853 and the dial-in number for international callers is 201-612-7415. To access the replay you will need the conference identification number, 13718346.

The conference call will also be webcast live and can be accessed at investor.incyte.com.

On May 4, 2021 Exelixis, Inc. (Nasdaq: EXEL) and GamaMabs Pharma SA reported that they have entered into an agreement under which Exelixis will, upon the future closing of the asset purchase and subject to certain conditions to closing, acquire all rights, title and interest in GamaMabs’ AMHR2 antibody technology (Press release, Exelixis, MAY 4, 2021, View Source [SID1234579096]). Exelixis will pay GamaMabs $5 million upon signing of the agreement, make additional payments upon completion of closing conditions, and make additional milestone payments after closing, contingent upon various events. Once the transfer is completed, Exelixis will control 100% of GamaMabs’ AMHR2 franchise technology including all assets pertaining to GamaMabs’ monoclonal antibody drug product murlentamab (GM-102).

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"GamaMabs has generated a compelling body of preclinical data supporting the potential of AMHR2 as a target for novel oncology therapies and demonstrated the safety of an anti-AMHR2 monoclonal antibody in human clinical trials," said Peter Lamb, Ph.D., Executive Vice President, Scientific Strategy and Chief Scientific Officer of Exelixis. "Based on these data, we believe that applying our ADC capabilities to GamaMabs’ panel of antibodies against AMHR2 could yield a promising new addition to our biotherapeutics portfolio. Acquiring GamaMabs’ extensive know-how related to this target, as well as existing drug product and related manufacturing cell lines, will allow us to reduce significantly the development timeline compared with starting an AMHR2 program de novo. This is consistent with our strategy of advancing novel cancer therapies as rapidly as possible in order to enable new treatment options that may provide improved patient benefit."

While AMHR2 expression is normally restricted to ovary, testis and adrenal tissues, it is also expressed in ovarian, endometrial, renal, liver, colon and lung tumors. Murlentamab, a monoclonal antibody targeting AMHR2, was well tolerated when administered on its own in Phase 1 and 2 studies, with no dose-limiting toxicities observed. In early 2021, GamaMabs discontinued development of murlentamab given the modest single-agent efficacy observed in these trials. Although it does not have plans to move murlentamab forward, Exelixis is encouraged by the potential of the AMHR2 target as it pursues the further discovery and development of novel biologics.

"The preclinical and clinical data generated to date for murlentamab support its first-in-class potential in a variety of cancer indications, and we expect that Exelixis’ ADC capabilities and expertise in the development and commercialization of novel cancer therapies will help to realize the full potential of this antibody in addressing unmet patient need," said Stéphane Degove, Chief Executive Officer at GamaMabs. "We believe that placing our AMHR2 franchise with Exelixis will enable rapid and effective development of AMHR2-targeting cancer therapies while providing us with near-term revenue that can support development of our other pipeline programs and technologies."

On May 4, 2021 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated agents that target the underlying mechanisms of cancer, reported financial results for the three months ended March 31, 2021 and provided a corporate update (Press release, Aptose Biosciences, MAY 4, 2021, View Source [SID1234579092]).

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The net loss for the quarter ended March 31, 2021 was $16.2 million ($0.18 per share) compared with $11.5 million ($0.15 per share) for the quarter ended March 31, 2020. Total cash and cash equivalents and investments as of March 31, 2021 were $112.1 million. Based on current operations, Aptose expects that cash on hand and available capital provide the Company with sufficient resources to fund all planned Company operations including research and development into the first half of 2023.

"In our ongoing Phase 1 a/b clinical trials with luxeptinib (formerly CG-806), we are encouraged by indicators of target engagement and anti-cancer activity, as well as a safety and tolerability profile that has allowed us to continue dose escalations," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "These hematologic malignancy patients represent highly challenging relapsed and refractory populations – the best currently available therapeutics have already failed these patients – and we are eager to see the effects of increasing luxeptinib drug exposures over a longer period of time. We look forward to providing an update for each of our clinical trials at the 2021 EHA (Free EHA Whitepaper) Virtual Congress on June 10-13th."

Separately, Aptose reported that Jotin Marango, M.D., Ph.D., has been appointed Chief Financial Officer, in addition to his Chief Business Officer role at Aptose. Dr. Marango brings more than a decade of finance, strategy and operations experience to the position, including several positions as biotechnology research analyst. "Dr. Marango is a highly accomplished biotech executive with financial acumen and a deep knowledge of capital markets and banking practices to oversee an established internal finance and accounting team, as well as an understanding of the scientific and medical underpinnings of cancer drugs," said Dr. Rice. "He brings Wall Street insight and the financial and strategic leadership necessary for this position at a time of significant opportunity and growth for Aptose."

Key Corporate Highlights

Luxeptinib Phase 1 a/b Clinical Study in AML – Luxeptinib is currently being evaluated in a Phase 1 a/b dose escalation clinical study in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML). Aptose recently reported anti-leukemic activity at the first dose level of 450 mg BID, and patients are now being treated at the second dose level of 600 mg BID. Updated data will be presented at EHA (Free EHA Whitepaper) in June. More information is available at www.clinicaltrials.gov (NCT04477291).

Luxeptinib Phase 1 a/b Clinical Study in B-cell Malignancies – In parallel with the trial in AML patients, luxeptinib is being evaluated in a Phase 1 a/b dose escalation clinical study in patients with relapsed or refractory B-cell malignancies, including chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphomas (NHL). Aptose is currently treating patients at the fifth dose level of 750 mg BID and enrolling additional patients at lower dose levels. Thus far, Aptose has observed on-target activity, including inhibition of multiple oncogenic driver kinases, treatment-related lymphocytosis, and tumor reductions. Updated data will be presented at EHA (Free EHA Whitepaper) in June. More information is available at www.clinicaltrials.gov (NCT03893682).

APTO-253 Phase 1 a/b Clinical Study in AML and MDS – As a direct inhibitor of MYC transcription, APTO-253 represents a novel approach for targeting MYC, an oncogene estimated to contribute to the majority of all human cancers, including hematologic malignancies. Aptose has completed the fifth cohort at the 150 mg/ m2 dose level of APTO-253, and has begun enrolling patients in the sixth dose cohort at 210 mg/m2. More information is available at www.clinicaltrials.gov (NCT02267863).

Aptose Continues to Expand the Leadership Team With Appointment of VP of Biometrics – Aptose announced the appointment of Dr. Yuying Jin to Vice President of Biometrics. Dr. Jin brings to Aptose broad experience in statistics, programming and data management, and is specialized in the development and execution of study design, hypothesis testing and statistical analyses for all phases of clinical trials, with a focus on oncology targeted therapy. Prior to joining Aptose in 2019, she served as a Program Head in Biostatistics at Intercept Pharmaceuticals and spent seven years working as a team lead and lead statistical reviewer at the US Food and Drug Administration (FDA), following a research position at the Fred Hutchinson Cancer Research Center. Dr. Jin holds a Ph.D. in biostatistics and an M.S. degree in statistics.
RESULTS OF OPERATIONS

A summary of the results of operations for the three-month periods ended March 31, 2021 and 2020 is presented below:

The net loss for the three-month period ended March 31, 2021 increased by $4.7 million to $16.2 million as compared with $11.5 million for the comparable period in 2020. Components of the net loss are presented below:

Research and Development
The research and development expenses for the three-month periods ended March 31, 2021 and 2020 were as follows:

Research and development expenses increased by $2.3 million to $8.2 million for the three-month period ended March 31, 2021 as compared with $5.9 million for the comparative period in 2020. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:

Program costs for luxeptinib increased by approximately $1 million, mostly as a result of the luxeptinib Phase 1 a/b AML trial, for which we received an IND allowance in June 2020, including higher manufacturing costs, costs to scale up manufacturing and research associated with formulation development, as well as higher clinical site costs.

Program costs for APTO-253 increased by approximately $211 thousand, mostly as a result of higher manufacturing and clinical trial costs related to the APTO-253 Phase 1b trial.

Personnel-related expenses increased by $485 thousand, mostly related to new positions hired to support our clinical trials and manufacturing activities.

Stock-based compensation increased by approximately $578 thousand in the three months ended March 31, 2021, compared with the three months ended March 31, 2020, mostly related to higher compensation expense in the current period on options issued in the first quarter of 2021.
General and Administrative
The general and administrative expenses for the three-month periods ending March 31, 2021 and 2020 were as follows:

General and administrative expenses for the three-month period ended March 31, 2021 were $8.0 million as compared with $5.9 million for the comparative period in 2020, an increase of approximately $2.1 million. The increase was primarily as a result of the following:

General and administrative expenses, other than share-based compensation and depreciation of equipment, increased by approximately $460 thousand in the three months ended March 31, 2021, primarily as a result of higher personnel related costs, higher insurance costs and higher office administrative costs offset by lower consulting fees and lower travel expenses.

Stock-based compensation increased by approximately $1.7 million in the three months ended March 31, 2021, compared with the three months ended March 30, 2020, mostly related to the modification of option agreements of one officer as part of a separation and release agreement. Vested options of 1,679,169 with exercise prices ranging from $1.03 to $7.44 were allowed to continue to be exercisable for an additional twelve-month period, and also 504,833 options that would have expired unvested, were allowed to continue to vest for a 12 month period. As there was no service requirement, the company recorded $945 thousand and $663 thousand additional compensation in the current period related to these modifications for the vested and unvested options, respectively.
Conference Call and Webcast

Aptose will host a conference call to discuss results for the quarter ended March 31, 2021 today, Tuesday, May 4, 2021 at 4:30 PM ET. Participants can access the conference call by dialing 1-844-882-7834 (North American toll-free number) and 1-574-990-9707 (international/toll number) and using conference ID # 2788133. The conference call can be accessed here and will also be available through a link on the Investor Relations section of Aptose’s website at View Source An archived version of the webcast along with a transcript will be available on the Company’s website for 30 days. An audio replay of the webcast will be available approximately two hours after the conclusion of the call for seven days by dialing 1-855-859-2056 (toll free number) and 1-404-537-3406 (international/toll number), using the conference ID # 2788133.

The press release, the financial statements and the management’s discussion and analysis for the quarter ended March 31, 2021 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

On May 4, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will present at the Bank of America 2021 Healthcare Conference at 11:00 a.m. Eastern Time on Tuesday May 11, 2021 (Press release, Neurocrine Biosciences, MAY 4, 2021, View Source [SID1234579091]). Kevin Gorman, Chief Executive Officer, will present at the conference.

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The live presentation will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

On May 4, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ending March 31, 2021 (Press release, PTC Therapeutics, MAY 4, 2021, View Source [SID1234579090]).

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"Overall PTC has had a strong performance this quarter through all aspects of the company from discovery to commercial revenue," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I would like to highlight the continued strong growth of the DMD franchise which has had one of our largest quarterly revenues to date. The other key milestone was the positive preliminary results in our PTC518 Huntington’s disease program demonstrating dose dependent lowering of the HTT mRNA. Analogous to the SMA program we are now well positioned with a clear path for success."

Key First Quarter and Other Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise had a total net product revenue of $90 million for Translarna (ataluren) and Emflaza (deflazacort) in the first quarter of 2021. This represents 32% growth over the first quarter of 2020 and one of PTC’s strongest quarterly commercial revenues to date.
Broader uptake due to new patients in existing geographies and geographic expansion drove Translarna growth.
Emflaza revenue growth was primarily due to increased new prescriptions, high compliance, and fewer discontinuations.
In March 2021, the European Medicines Agency (EMA) approved Evrysdi (risdiplam) in the European Union (EU). The first sale of Evrysdi in this region was recorded the following day, triggering a $20 million milestone payment to PTC. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
Preliminary results from the PTC518 Phase 1 healthy volunteer trial demonstrated dose-dependent reduction of Huntington mRNA beyond the 30-50% target.
PTC received Gallup’s Don Clifton Strengths-Based Culture Award, which reflects the Company’s ongoing deep commitment to its employees.
First Quarter Clinical Updates:

PTC has multiple clinical trials ongoing, three of which are registration-directed clinical studies:
The MIT-E Phase 2/3 trial with vatiquinone for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 trial with vatiquinone for Friedreich ataxia with data anticipated in 2023.
The FITE19 Phase 2/3 clinical trial for PTC299 in patients with COVID-19 with an expected data readout in the second half of 2021.
The second Bio-e compound, PTC857 healthy volunteer study was recently completed, and data will be communicated in the second quarter.
The registration-directed Phase 3 PTC923 phenylketonuria (PKU) trial, APHENITY, is expected to initiate in mid-2021.
The Committee for Medicinal Products for Human Use (CHMP) has requested a clock stop in the aromatic L-amino acid decarboxylase (AADC) deficiency review process to allow for completion of its pre-approval inspections, which were delayed due to COVID-19. The CHMP opinion is now anticipated in the third quarter of 2021.
Due to COVID-related surgical delays, the AADC-deficiency biologics license application submission to the U.S. Food and Drug Administration is anticipated to be delayed by at least one quarter.
First Quarter 2021 Financial Highlights:

Total revenues were $117.9 million for the first quarter of 2021, compared to total revenues of $68.3 million for the first quarter of 2020, a 32% increase. Total revenue includes net product revenue of $91.3 million and collaboration and royalty revenue of $26.7 million in the first quarter of 2021.
Translarna net product revenues were $46.5 million for the first quarter of 2021, compared to $40.5 million for the first quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion into new territories.
Emflaza net product revenues were $43.5 million for the first quarter of 2021, compared to $27.5 million for the first quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
Roche reported Evrysdi first quarter 2021 sales of approximately CHF 80 million. During the first quarter of 2021, the first commercial sale of Evrysdi in the EU triggered a $20 million milestone payment to PTC, which was reported as collaboration revenue.
U.S. GAAP (generally accepted accounting principles) R&D expenses were $134.5 million for the first quarter of 2021, compared to $90.1 million for the first quarter of 2020. The increase in R&D expenses reflects costs associated with increased investment in research programs, and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $120.8 million for the first quarter of 2021, excluding $13.7 million in non-cash, stock-based compensation expense, compared to $81.9 million for the first quarter of 2020, excluding $8.2 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $61.1 million for the first quarter of 2021, compared to $58.2 million for the first quarter of 2020. The increase in SG&A expenses was associated with entering into a long-term lease for the Hopewell facility that commenced on July 1, 2020.
Non-GAAP SG&A expenses were $49.1 million for the first quarter of 2021, excluding $12.0 million in non-cash, stock-based compensation expense, compared to $51.2 million for the first quarter of 2020, excluding $7.0 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $0.1 million for the first quarter of 2021, compared to $0.9 million for the first quarter of 2020. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
Net loss was $128.6 million for the first quarter of 2021, compared to net loss of $112.7 million for the first quarter of 2020.
Cash, cash equivalents and marketable securities was $988.4 million at March 31, 2021, compared to $1.1 billion at December 31, 2020.
Shares issued and outstanding as of March 31, 2021 were 70,405,905.
PTC Reaffirms Full Year 2021 Guidance as Follows:

PTC anticipates net product revenues for the DMD franchise for the full year 2021 to be between $355 and $375 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 million.
Non-GAAP Financial Measures:

In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the Company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.

Today’s Conference Call and Webcast Reminder:

PTC will host a conference call to discuss the first quarter of 2021 corporate updates and financial results today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 4292410. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.