On May 4, 2021 BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, reported that the company will present virtually at the 2021 RBC Capital Markets Global Healthcare Conference on Thursday, May 20th, 2021 at 12:05 pm ET (Press release, BioTheryX, MAY 4, 2021, View Source [SID1234579129]).

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Rob Williamson, President and CEO, will present an overview on the company’s targeted protein degradation platform, its clinical stage oncology pipeline, and the management team will participate in 1x1s with investors.

On May 4, 2021 HUTCHMED (China) Limited (Nasdaq/AIM: HCM), formerly known as Hutchison China MediTech Limited, reported that, further to its "Results of Annual General Meeting" announcement released on Wednesday, April 28, 2021, the Registrar of Companies in the Cayman Islands has issued a replacement certificate of incorporation on Thursday, April 29, 2021 in the name of HUTCHMED (China) Limited ("HUTCHMED") so the new name of the Company is now effective from April 29, 2021 (Press release, Hutchison China MediTech, MAY 4, 2021, View Source [SID1234583631]).

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Trading in the shares of HUTCHMED will commence under the new name HUTCHMED (China) Limited with effect from 8:00 am UK time on Wednesday, May 5, 2021. The stock market ticker symbol, depositary interests ISIN and American depositary shares’ ISIN and CUSIP of the Company remain unchanged.

As disclosed in the 2020 final results announcement, the website of the Company has already changed to www.hutch-med.com with effect from March 4, 2021. The information required pursuant to AIM Rule 26 is available at this website.

This name change is a consolidation of the two corporate identities that we have used since our inception, under a single and ubiquitous corporate identity that captures the history and brand equity we have built over the past twenty years. For more information, please visit www.hutch-med.com/chi-med-becomes-hutchmed.

Shareholders should note that their shareholding will not be affected by the change of name of the Company and the existing share certificates should be retained as they will remain valid for all purposes and no new share certificates will be issued.

The change of name was approved at the annual general meeting held on Wednesday, April 28, 2021.

On May 4, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that Ellen Kim, MD, Medical Director, Dermatology Clinic, Perelman Center for Advanced Medicine, Professor of Dermatology at the Hospital of the University of Pennsylvania, and the Lead Principal Investigator for the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study, presented confirmatory data at the Society for Investigative Dermatology (SID) Virtual Meeting, held May 3-8, 2021 (Press release, Soligenix, MAY 4, 2021, View Source [SID1234579044]). The presentation was selected to be shown during a Concurrent Mini-Symposium for Patient-Targeted Research. The presented data demonstrated the ability of HyBryte (SGX301) to treat both patch and plaque disease, including generating complete disease responses, while being associated with fewer and less severe adverse events than other currently approved skin-directed therapies for cutaneous T-cell lymphoma (CTCL).

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Oral Presentation:

Topical hypericin ointment photodynamic therapy is effective and safe in CTCL (FLASH study) Dr. Kim’s presentation is archived on the SID Virtual Meeting site and will be accessible via registration until May 31, 2021. The abstract is available here and attendees can register to see the presentation here.

Key Highlights:

HyBryte is activated by visible light at a wavelength of 500-650 nm, which provides deeper dermal penetration than ultraviolet (UV) spectrum light. This resulted in statistically significant clinical responses observed in patches as well as deeper plaque lesions, which are typically more difficult to treat and generally less responsive to UV light therapy.
In addition to its demonstrated, statistically significant efficacy which ultimately led to 49% of patients achieving at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) after 18 weeks of therapy (p<0.0001), complete responses of all treated index lesions were also shown to occur. These complete responses increased in frequency as treatment with HyBryte continued and photographs demonstrating this response were reviewed.
Compared to other, second-line, approved drugs for the treatment of CTCL, HyBryte demonstrated significantly less safety concerns. This was reflected in the low rate of study discontinuation attributed to adverse events which showed only a 5% overall drop-out rate during the treatment phase in HyBryte treated patients, lower than typically observed in other early stage CTCL trials.
"Additional CTCL therapies with fewer and less severe side effects are desperately needed in our field," noted Dr. Kim. "I believe the efficacy and safety we saw HyBryte demonstrate in this trial proves that it can help us fill that need. This study was the largest multicenter, randomized, double-blind, placebo-controlled skin directed therapy study in MF/CTCL (mycosis fungoides/cutaneous T-cell lymphoma) to date and I would like to thank all site investigators, and especially our patients, for their contributions to developing this promising new therapy."

About the Society for Investigative Dermatology Virtual Meeting

The SID meeting is an annual meeting, dedicated to a broad range of dermatology related topics, as described here. SID’s mission is to advance the sciences relevant to skin diseases through education, advocacy and scholarly exchange of scientific information.

About HyBryte

HyBryte (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.

On May 4, 2021 Cerus Corporation (Nasdaq: CERS) reported financial results for the first quarter ended March 31, 2021 (Press release, Cerus, MAY 4, 2021, View Source [SID1234579071]).

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Recent developments and highlights include:

First quarter 2021 total revenue of $29.6 million, reflecting a 20% increase over the prior year period. Total revenue was composed of (in millions, except %):

Increasing 2021 annual product revenue guidance range to $110 million to $114 million (from prior guidance of $106 million to $110 million), representing an approximately 20% to 24% increase over full year 2020 reported product revenue.
Multiple blood center partners have completed validation efforts and are now manufacturing INTERCEPT Fibrinogen Complex, with inventory ready for release to hospital customers.
The Centers for Medicare and Medicaid Services (CMS) published the Fiscal Year 2022 Hospital Inpatient Prospective Payment System (IPPS) Proposed Rule, which recommended that Intercept Fibrinogen Complex be eligible for incremental payment via a New Technology Add-On Payment (NTAP) when performed in the hospital inpatient setting.
Third module (CMC Data) submitted for CE Mark for INTERCEPT red blood cells. Fourth and final module (Manufacturing) submission anticipated by end of Q2:21.
Cash, cash equivalents, and short-term investments of $132 million at March 31, 2021.
"On the heels of our strong 2020 performance, product revenue in the first quarter of $23.4 million exceeded our expectations," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "As our U.S. customers continue to prepare for compliance with FDA’s guidance around the bacterial safety of platelets, demand for the INTERCEPT Blood System continues to build. The momentum we have in the U.S. market in particular gives us confidence that 2021 will be another year of strong growth for Cerus and we are increasing our full year product revenue guidance accordingly."

Revenue

Product revenue during the first quarter of 2021 was $23.4 million, compared to $18.6 million during the same period in 2020. Product revenue growth during the quarter benefited from increased demand for INTERCEPT platelet products in the U.S., led by adoption from the top five blood center networks in the country.

First quarter government contract revenue was $6.2 million, compared to $6.0 million during the same period in 2020. First quarter government contract revenue is primarily comprised of funding from the Biomedical Advanced Research and Development Authority (BARDA) agreement for the development of the INTERCEPT Blood System for Red Cells. The total potential value of the current BARDA agreement is $214 million, with $72.7 million cumulatively invoiced to BARDA through March 31, 2021.

BARDA is part of the Office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services. The development of the INTERCEPT red blood cell program has been funded in whole or in part with Federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority, under Contract No. HHSO100201600009C.

Gross Margins

Gross margins on product revenue during the first quarter of 2021 were 52.5% compared to 55.3% for the first quarter of 2020. The decrease in gross margin was tied to increased sales to our U.S. customers, who typically use our single dose platelet kits, which carry a less favorable gross margin contribution compared to our double dose kits, which are used more broadly outside of the U.S.

Operating Expenses

Total operating expenses for the first quarter of 2021 were $34.9 million compared to $31.7 million for the same period of the prior year.

Selling, general, and administrative (SG&A) expenses for the first quarter of 2021 totaled $19.2 million, compared to $15.9 million for the first quarter of 2020. The year-over-year increase in SG&A expenses was tied to incremental expenses associated with the launch of our INTERCEPT Fibrinogen Complex product as well as increased non-cash stock-based compensation expense.

Research and development (R&D) expenses for the first quarter of 2021 were $15.7 million, compared to $15.8 million for the first quarter of 2020. The Company continues to expect to incur costs associated with the development and testing of a new generation of INTERCEPT devices and disposable kits as well as the clinical development and pursuit of regulatory approval for the red blood cell system, among others.

Net Loss

Net loss for the first quarter of 2021 was $17.5 million, or $0.10 per basic and diluted share, compared to a net loss of $16.5 million, or $0.10 per basic and diluted share, for the first quarter of 2020. Non-cash stock-based compensation was higher by approximately $1.6 million during the first quarter of 2021 compared to the prior year period, contributing to the slightly higher net loss.

Balance Sheet

At March 31, 2021, the Company had cash, cash equivalents and short-term investments of $131.7 million, compared to $133.6 million at December 31, 2020.

Despite continued increased investments in inventory to meet customer demand, cash used for operations during Q1 2021 was narrower at $18.0 million compared to $19.8 million used during the prior year period. Offsetting this lower use were $15 million in term loan draws and $1.4 million in incremental draws from its revolving line of credit. At March 31, 2021, the Company had approximately $54.6 million in outstanding term loan debt and $9.9 million of borrowings under its revolving loan credit agreement, compared to $39.6 million in outstanding term loan debt and $8.5 million of borrowings under its revolving loan credit agreement at December 31, 2020.

Increasing 2021 Product Revenue Guidance

The Company now expects 2021 product revenue to be in the range of $110 million to $114 million, as compared to the prior range of $106 million to $110 million. The revised guidance range represents approximately 20% to 24% growth compared to 2020 reported product revenue.

Quarterly Conference Call

The Company will host a conference call at 4:30 P.M. EDT this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on Cerus’ website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 8668508. The replay will be available approximately three hours after the call through May 18, 2021.

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.