Kineta Closes Investment Round to Advance Early Preclinical Development of Immuno-oncology Programs

On May 5, 2020 Kineta, Inc., a clinical stage biotechnology company focused on the development of novel immunotherapies in oncology, neuroscience and biodefense, reported that they have successfully closed their most recent funding round totaling $5 million (Press release, Kineta, MAY 5, 2020, View Source;utm_medium=rss&utm_campaign=kineta-closes-investment-round-advance-immuno-oncology [SID1234557002]). This round was led by the Bellevue-based Schlaepfer Family Foundation.

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"Our team is thrilled to quickly close this round with significant investments from the Schlaepfer Family Foundation as well as current investors," said Shawn Iadonato, Chief Executive Officer of Kineta. "This funding will allow us accelerate the development of our novel immuno-oncology programs and enable our mission to develop innovative immunotherapies that transform patients’ lives".

"We appreciate Kineta’s commitment to developing breakthrough therapies for cancer patients, which personally touches so many of our lives," said Phil Scott Schlaepfer, co-founder of the Schlaepfer Family Foundation. "Our foundation is proud to invest in this local biotech company and their experienced team of scientists. We recognized Kineta’s excellence in both scientific and business execution several years ago when we made our initial investment. The team at Kineta continues to be a global leader in advancing life-saving research."

Proceeds from this investment round will be used to fund the early development of Kineta’s immuno-oncology drug programs. Kineta is focused on developing new, best-in-class immunotherapies to address hard-to-treat cancers in a variety of solid tumors.

Selecta Biosciences to Present at the Bank of America Merrill Lynch 2020 Health Care Conference

On May 5, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, reported that Selecta’s Chief Executive Officer, Carsten Brunn, Ph.D., will present at the Bank of America Merrill Lynch 2020 Health Care Conference, which is being held virtually, on Tuesday, May 12 at 5:00 p.m. Eastern Time (Press release, Selecta Biosciences, MAY 5, 2020, View Source [SID1234557001]).

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A live webcast and a copy of the presentation will be available on the Investors & Media section of the Selecta website at www.selectabio.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 90 days.

Incyte Reports 2020 First Quarter Financial Results and Provides Updates on Key Clinical Programs

On May 5, 2020 Incyte (Nasdaq: INCY) reported 2020 first quarter financial results, and provides a status update on the Company’s development portfolio (Press release, Incyte, MAY 5, 2020, View Source [SID1234556999]).

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"Our strong first quarter results highlight continued revenue momentum, led by robust demand across all three indications for Jakafi (ruxolitinib)," stated Hervé Hoppenot, Chief Executive Officer, Incyte. "As we navigate this unprecedented and uncertain time, we have implemented numerous initiatives as we seek to ensure continuity of patient care. We are continuing to execute on our goals, and we were very pleased to announce the FDA approval of Pemazyre (pemigatinib), the first of three potential product approvals that we expect to announce in 2020. The FDA review of the capmatinib NDA and tafasitamab BLA are proceeding as expected and, following positive results from our Phase 3 TRuE-AD development program in atopic dermatitis, we are also on track to submit the NDA for ruxolitinib cream at the end of 2020, all of which positions us for what I expect to be a transformational year."

COVID-19

Commercial, Supply and Clinical & Regulatory Impact

While it is currently not possible to predict the overall long-term impact of the COVID-19 pandemic on Incyte’s business, to-date, there has been no impact on the commercial side of the business, and Incyte currently has ample commercial and clinical supply of our medicines to meet the needs of patients receiving Incyte’s approved medicines and those participating in global clinical trials. Incyte’s manufacturing processes are proceeding as usual, with increased manufacturing efforts for ruxolitinib in place to respond to the COVID-19 pandemic and study requests. Incyte continues to move forward with its global clinical trials, and late-stage programs remain broadly on track, although short-term effects may continue to emerge. For example, while ongoing monitoring of already-enrolled patients is expected to continue, new patient recruitment in certain clinical studies may be impacted. The impact on clinical trials may also vary by disease state and by severity of disease, as well as by geography, as some regions are more adversely impacted.

Ruxolitinib and baricitinib as potential therapies for patients with COVID-19

Incyte has initiated a Phase 3 clinical trial (RUXCOVID) to evaluate the efficacy and safety of ruxolitinib plus standard-of-care (SoC), compared to SoC therapy alone, in patients with COVID-19 associated cytokine storm. The collaborative study is sponsored by Incyte in the United States and by Novartis outside of the United States.

Incyte is also opening a second Phase 3 clinical trial in the United States to evaluate the efficacy and safety of ruxolitinib plus SoC, compared to SoC therapy alone, in COVID-19 patients on mechanical ventilation and who have acute respiratory distress syndrome (ARDS), a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs.

Additionally, Incyte has launched an emergency Expanded Access Program in the United States to allow eligible patients with COVID-19 associated cytokine storm to receive ruxolitinib.

In April, Lilly announced it has entered into an agreement with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), to study baricitinib as an arm in NIAID’s Adaptive COVID-19 Treatment Trial. The study will investigate the efficacy and safety of baricitinib as a potential treatment for hospitalized patients diagnosed with COVID-19 in the US, and Lilly is also planning a study expansion to include Europe and Asia.

Portfolio Update

LIMBER – key highlights

Key LIMBER development programs, including the once-a-day (QD) formulation of ruxolitinib, and the ongoing and planned ruxolitinib combinations with parsaclisib (PI3Kδ), PIM, BET and ALK2 are currently on track.

Indication and status

Once-a-day ruxolitinib
(JAK1/JAK2)


Myelofibrosis and polycythemia vera: clinical pharmacology studies

ruxolitinib + parsaclisib
(JAK1/JAK2 + PI3Kδ)


Refractory myelofibrosis: Phase 3 in preparation

ruxolitinib + INCB53914
(JAK1/JAK2 + PIM)


Refractory myelofibrosis: Phase 2

ruxolitinib + INCB57643
(JAK1/JAK2 + BET)


Refractory myelofibrosis: Phase 2 in preparation

ruxolitinib + INCB00928
(JAK1/JAK2 + ALK2)


Myelofibrosis: Phase 2 in preparation

Oncology beyond MPNs – key highlights

In April, the FDA approved Pemazyre, Incyte’s selective fibroblast growth factor receptor (FGFR) inhibitor, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement as detected by an FDA-approved test. Pemazyre was reviewed under Breakthrough Therapy designation and granted accelerated approval based on overall response rate and duration of response. The marketing authorization application (MAA) seeking approval for pemigatinib in Europe is under review by the European Medicines Agency (EMA).

Delays in data collection and validation, caused by COVID-19 related disruption, mean that data from the FIGHT-201 clinical trial of pemigatinib in patients with bladder cancer are now expected to be presented at a medical conference in early 2021; all other clinical trials of pemigatinib are currently progressing as expected.

Data from the Phase 3 REACH 2 trial of ruxolitinib versus best available therapy (BAT) in patients with steroid-refractory acute graft-versus-host disease (GVHD) have been published in The New England Journal of Medicineand are also expected to be presented at the 46th annual meeting of the European Society for Blood and Marrow Transplantation (EBMT) in August 30-September 2, 2020 (postponed from March 22-25, 2020). The Phase 3 REACH 3 trial of ruxolitinib versus BAT in patients with steroid-refractory chronic GVHD has completed recruitment and results are expected in the second half of 2020.

The collaboration and license agreement with MorphoSys for the development and commercialization of tafasitamab became effective in March. In February, the FDA granted Priority Review for tafasitamab in combination with lenalidomide for the treatment of relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL), and set a PDUFA goal date of August 30, 2020.

Preparations are ongoing to initiate the Phase 3 POD1UM-304 trial of retifanlimab (formerly INCMGA0012) in combination with platinum-based chemotherapy as a first-line treatment for patients with non-small cell lung cancer (NSCLC). Incyte no longer plans to pursue the Phase 3 POD1UM-301 trial of retifanlimab in combination with chemoradiation therapy (CRT) in participants with unresectable, Stage III NSCLC.


Indication and status

ruxolitinib
(JAK1/JAK2)


Steroid-refractory chronic GVHD: Phase 3 (REACH3)1

itacitinib
(JAK1)


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

pemigatinib
(FGFR1/2/3)


Cholangiocarcinoma: Phase 2 (FIGHT-202), Phase 3 (FIGHT-302); MAA under review
Bladder cancer: Phase 2 (FIGHT-201, FIGHT-205)
8p11 MPN: Phase 2 (FIGHT-203)
Tumor agnostic: Phase 2 (FIGHT-207)

tafasitamab

(CD19)2


r/r DLBCL: Phase 2 (L-MIND); Phase 3 (B-MIND); BLA under review
1L DLBCL: Phase 1b (First-MIND)

parsaclisib
(PI3Kδ)


Follicular lymphoma: Phase 2 (CITADEL-203)
Marginal zone lymphoma: Phase 2 (CITADEL-204)
Mantle cell lymphoma: Phase 2 (CITADEL-205)

retifanlimab
(PD-1)3


MSI-high endometrial cancer: Phase 2 (POD1UM-101)
Merkel cell carcinoma: Phase 2 (POD1UM-201)
Anal cancer: Phase 2 (POD1UM-202)
NSCLC: Phase 3 (POD1UM-304) in preparation

1) Clinical development of ruxolitinib in GVHD conducted in collaboration with Novartis
2) Development of tafasitamab in collaboration with MorphoSys
3) Retifanlimab licensed from MacroGenics

Inflammation and Autoimmunity (IAI) – key highlights

In April, safety and efficacy data from the two Phase 3 trials in the TRuE-AD program evaluating ruxolitinib cream in mild-to-moderate atopic dermatitis were presented at the Revolutionizing Atopic Dermatitis (RAD) virtual symposium. The 44-week long-term safety and efficacy portion of both the TRuE-AD1 and TRuE-AD2 trials are ongoing and the NDA submission is expected before the end of 2020.

The two Phase 3 trials in the TRuE-V pivotal program evaluating ruxolitinib cream in patients with vitiligo are currently proceeding as planned, and results are expected in 2021.


Indication and status

ruxolitinib cream
(JAK1/JAK2)


Atopic dermatitis: Phase 3 (TRuE-AD1, TRuE-AD2; primary endpoints met)
Vitiligo: Phase 3 (TRuE-V1, TRuE-V2)

INCB54707
(JAK1)


Hidradenitis suppurativa: Phase 2

parsaclisib
(PI3Kδ)


Autoimmune hemolytic anemia: Phase 2

INCB00928
(ALK2)


Fibrodysplasia ossificans progressiva: Phase 2 in preparation

Discovery and early development – key highlights

Based on emerging data from the FGFR4 inhibitor program, development of INCB62079 has been discontinued because of insufficient efficacy in the target patient population. Incyte’s portfolio of other earlier-stage clinical candidates is summarized below.

Modality


Candidates

Small molecules


INCB01158 (ARG)1, INCB81776 (AXL/MER), epacadostat (IDO1), INCB59872 (LSD1), INCB86550 (PD-L1)

Monoclonal antibodies2


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

Bispecific antibodies


MCLA-145 (PD-L1xCD137)3

1) INCB01158 development in collaboration with Calithera
2) Discovery collaboration with Agenus
3) MCLA-145 development in collaboration with Merus

Partnered – key highlights

In March, Incyte and Lilly announced the FDA has granted Breakthrough Therapy designation for baricitinib for the treatment of alopecia areata, an autoimmune disorder that can cause unpredictable hair loss on the scalp, face and other areas of the body. In January, Lilly submitted baricitinib for regulatory review in Europe and Japan as a treatment for patients with moderate-to-severe atopic dermatitis, and has announced plans to submit for approval in the U.S. in 2020.

In February, Incyte and Novartis announced that the NDA for capmatinib, seeking approval in patients with metastatic non-small cell lung cancer (NSCLC) and with a mutation leading to exon 14 skipping as detected by an FDA-approved test, was accepted for Priority Review by the FDA.


Indication and status

baricitinib (JAK1/JAK2)1


Atopic dermatitis: Phase 3 (BREEZE-AD)
Systemic lupus erythematosus: Phase 3
Severe alopecia areata: Phase 3 (BRAVE-AA1)

capmatinib (MET)2


NSCLC (with MET exon 14 skipping mutations): NDA (by Novartis) under review

1) Worldwide rights to baricitinib licensed to Lilly: approved as Olumiant in multiple territories globally for certain patients with moderate-to-severe rheumatoid arthritis
2) Worldwide rights to capmatinib licensed to Novartis

2020 First Quarter Financial Results

The financial measures presented in this press release for the three months ended March 31, 2020 and 2019 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, 2020 increased 24% over the prior year comparative period as a result of increases in Jakafi and Iclusig net product revenues and higher product royalty revenues from Jakavi and Olumiant. Jakafi net product revenues for the quarter ended March 31, 2020 increased 22% over the prior year comparative period, primarily driven by growth in patient demand across all indications.

Operating Expenses
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.
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, 2020 increased 301% and 335%, respectively, compared to the same period in 2019, primarily due to upfront consideration of $805 million related to our collaborative agreement with MorphoSys.

Selling, general and administrative expenses GAAP and Non-GAAP selling, general and administrative expenses for the quarter ended March 31, 2020 decreased 10% and 12%, respectively, compared to the same period in 2019, primarily due to the timing of certain expenses.

Other Financial Information

Operating income (loss) GAAP and Non-GAAP operating income (loss) for the quarter ended March 31, 2020 decreased compared to the same period in 2019 primarily due to upfront consideration related to our collaborative agreement with MorphoSys, partially offset by the growth in product and royalty revenues.

Cash, cash equivalents and marketable securities position As of March 31, 2020 and December 31, 2019, cash, cash equivalents and marketable securities totaled $1.3 billion and $2.1 billion, respectively. The decrease reflects the upfront payment and stock purchase related to our collaborative agreement with MorphoSys.

2020 Financial Guidance

The Company has reaffirmed its full year 2020 financial guidance, as detailed below. The R&D expense guidance now includes the expenses related to Incyte’s share of tafasitamab development costs under the MorphoSys collaboration, but excludes $805 million of upfront consideration paid under the MorphoSys collaboration. The financial guidance also excludes 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 change in fair value of estimated future royalties relating to sales of Iclusig in the licensed territory relating to the acquisition of the European business of ARIAD Pharmaceuticals, Inc.

Future Non-GAAP financial measures may also exclude impairment of goodwill or other assets, changes in the fair value of equity investments in our collaboration partners, non-cash interest expense related to the amortization of the initial discount on our 2020 Senior Notes and the impact on our tax provision of discrete changes in our valuation allowance position on deferred tax assets.

Conference Call and Webcast Information

Incyte will hold a conference call and webcast this morning at 8:00 a.m. EDT. 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, 13702083.

If you are unable to participate, a replay of the conference call will be available for 30 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, 13702083.

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

About Incyte

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics.

For additional information on Incyte, please visit Incyte.com and follow @Incyte.

About Jakafi (ruxolitinib)

Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

Jakafi is also indicated for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea as well as adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

About Pemazyre (pemigatinib)

Pemazyre is a kinase inhibitor indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

Pemazyre is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3 which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations.

Pemazyre is marketed by Incyte in the United States. Incyte has granted Innovent Biologics, Inc. rights to develop and commercialize pemigatinib in hematology and oncology in Mainland China, Hong Kong, Macau and Taiwan. Incyte has retained all other rights to develop and commercialize pemigatinib outside of the United States.

Additionally, Incyte’s marketing authorization application (MAA) seeking the approval of pemigatinib for patients with cholangiocarcinoma in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that is relapsed or refractory after at least one line of systemic therapy.

Pemazyre is a trademark of Incyte Corporation.

About Iclusig (ponatinib) tablets

Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

In the EU, Iclusig is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation, or the treatment of adult patients with Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

Incyte has an exclusive license from ARIAD Pharmaceuticals, Inc., since acquired by Takeda Pharmaceutical Company Limited, to develop and commercialize Iclusig in the European Union and 22 other countries, including Switzerland, Norway, Turkey, Israel and Russia.

Karyopharm Reports First Quarter 2020 Financial Results and Highlights Recent Company Progress

On May 5, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an innovation-driven pharmaceutical company, reported financial results for the quarter ended March 31, 2020 (Press release, Karyopharm, MAY 5, 2020, View Source [SID1234556998]). In addition, Karyopharm highlighted select corporate milestones, including details regarding the ongoing U.S. commercialization of XPOVIO (selinexor), and provided an overview of its key clinical development programs.

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"I am extremely proud of the significant progress we have made thus far in 2020. Following our recent announcement of the positive top-line results from the pivotal Phase 3 BOSTON study, we are actively preparing to share the dataset at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 (ASCO20) Virtual Scientific Program and expect to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) by the end of May 2020, requesting an expansion of the currently approved indication for XPOVIO to include second line treatment for patients with relapsed or refractory multiple myeloma," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "Additionally, despite industry-wide challenges created by the current COVID-19 pandemic, we remain pleased by the ongoing commercialization of XPOVIO in the U.S., as well as our ability to significantly strengthen our balance sheet in the first quarter. Finally, I want to assure our many stakeholders that the entire Karyopharm team has risen to the challenge of navigating the Company through these difficult times and we are working day and night to provide critical medicines to the patients and physicians who need them most. In addition to the applications in oncology, we remain highly encouraged by the potential anti-viral and anti-inflammatory activity of XPO1 inhibition with selinexor and look forward to working, as quickly as possible, with the medical community of regulators, treating physicians and patients on advancing our new clinical study of low dose selinexor to treat patients with severe COVID-19."

First Quarter 2020 and Recent Highlights

XPOVIO in Multiple Myeloma

Pivotal Phase 3 BOSTON Study Met Primary Endpoint. In early March 2020, Karyopharm announced the top-line results from the BOSTON study, including that BOSTON met its primary endpoint of a statistically significant increase in progression-free survival (PFS). The BOSTON study is evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (bortezomib) (once weekly) and low dose dexamethasone (SVd), compared to standard twice weekly Velcade and low dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy. The median PFS in the SVd arm was 13.93 months compared to 9.46 months in the Vd arm, representing a 4.47 month (47%) increase in median PFS (hazard ratio=0.70; p=0.0066). There were no new safety signals on the SVd arm and there was no imbalance in deaths between the two arms in the study. The clinical data will be disclosed in an oral presentation at the ASCO (Free ASCO Whitepaper) 2020 Virtual Scientific Program taking place May 29-31, 2020. Karyopharm expects to submit an sNDA to the FDA by the end of May 2020 requesting expansion of the currently approved indication for XPOVIO to include second line treatment for patients with relapsed or refractory multiple myeloma. A regulatory submission to the European Medicines Agency (EMA) is also planned.

XPOVIO U.S. Commercialization. Oral XPOVIO became commercially available to patients in the U.S. in July 2019 and generated net product sales of $16.1 million in the first quarter of 2020. As of March 31, 2020, more than 2,200 XPOVIO prescriptions have been filled since launch, and more than 150 new physician prescribing accounts were added in the first quarter. Prescription refills for XPOVIO among existing patients remained strong in the quarter. Based on data from specialty pharmacies, approximately 60% of eligible patients have received a refill for their second prescription since launch, and the per patient average number of prescriptions continues to increase each quarter. Additionally, for those patients responding well to therapy, we continue to see an increasing and meaningful number of patients who have now been on therapy for five months or longer, further supporting the potential for longer-term use of XPOVIO therapy, as appropriate. We did experience a decrease in net product sales in the first quarter of 2020 compared to the fourth quarter of 2019, largely driven by minimal channel inventory build in the first quarter. Finally, there were fewer new patient XPOVIO starts than expected in the quarter, primarily due to the diversion of physician and other healthcare resources to manage the ongoing COVID-19 pandemic and the significant reduction in Karyopharm’s field force in-person activities as a result of shelter-in-place orders across the U.S. However, we were encouraged to see an acceleration in demand for XPOVIO in April 2020.

Decision from EMA for Marketing Authorization Application (MAA) Expected in 2020. In January 2019, Karyopharm submitted an MAA to the EMA requesting conditional approval for selinexor in combination with dexamethasone as a new treatment for patients with heavily pretreated multiple myeloma based on the results of the Phase 2b STORM study. In January 2020, Karyopharm was granted a three-month extension from the EMA’s Committee for Medicinal Products for Human Use (CHMP) to provide additional time to respond to the outstanding questions from the MAA, primarily re-monitoring certain clinical data. Due to the COVID-19 pandemic and the resulting disruption at many clinical sites, re-monitoring activities requested by CHMP remain ongoing. The Company does not anticipate any significant changes to the data sets supporting the MAA filing from the ongoing re-monitoring activities. The Company now expects to be able to respond to CHMP in mid-2020 and to receive a decision on the selinexor MAA by late-2020.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

FDA Accepts sNDA and Grants Priority Review. The FDA accepted for filing with Priority Review Karyopharm’s sNDA seeking accelerated approval for selinexor for the treatment of adult patients with relapsed or refractory DLBCL, not otherwise specified, who have received at least two prior therapies. The FDA assigned an action date of June 23, 2020 under the Prescription Drug User Fee Act (PDUFA). The Company also expects to submit an MAA to the EMA in 2021 requesting conditional approval for selinexor in the same indication. In addition to Orphan Drug Designation, selinexor was granted Fast Track designation for this indication by the FDA in 2018.
Low Dose Selinexor in COVID-19

First Patient Dosed in Randomized Study. Karyopharm recently announced the initiation of a new study to evaluate low dose oral selinexor in hospitalized patients with severe COVID-19. SINE compounds, including both selinexor and it’s close relative verdinexor, have been identified as having the potential to interfere with key host protein interactions with influenza, RSV and other viruses, including SARS-CoV-2, the virus that causes COVID-19. Furthermore, XPO1 (also called CRM1) was identified as one of the host proteins with the highest number of functional connections with SARS-CoV proteins. Recent preclinical experiments have further demonstrated selinexor’s ability to inhibit the viral propagation of the SARS-CoV-2 virus in monkey Vero cells, which are commonly used to study human viral infections. Finally, SINE compounds, including selinexor, have demonstrated potent anti-inflammatory activity through the inhibition of Nuclear Factor kB (NF-kB), leading to reductions in cytokines such as IL6, IL1, IFNγ and others in a variety of models. Since high levels of these cytokines are found in patients with COVID-19 and the most severe disease, reductions in these cytokines may be particularly beneficial to hospitalized patients with COVID-19. Patient enrollment and dosing has begun in this randomized, placebo-controlled, Phase 2 study (XPORT-CoV-1001 / NCT04349098), which is expected to enroll approximately 230 patients at clinical sites in the U.S., Europe, and Israel.
Corporate and Financial Updates

Expanded Territory Rights with Antengene Corporation and Reacquisition of Rights from Ono Pharmaceutical Co., Ltd. Karyopharm’s license agreement with Antengene, our current partner in China, was amended to provide Antengene with the exclusive right to develop and commercialize selinexor and eltanexor in all human oncology indications in Australia, New Zealand, South Korea, Taiwan, Hong Kong and the ASEAN countries. The amended agreement also includes the development and commercialization of KPT-9274 in all human oncology indications and verdinexor in human non-oncology indications in Australia and New Zealand. Under the terms of the amended agreement, Karyopharm will receive a one-time upfront payment of $12.0 million from Antengene, expected in the second quarter of 2020. Karyopharm is eligible to receive additional payments if certain future prespecified regulatory and commercial milestones are achieved by Antengene. Karyopharm is also eligible to receive tiered double-digit royalties based on future net sales of selinexor and eltanexor, and tiered single-to double-digit royalties based on future net sales of verdinexor and KPT-9274 in the expanded territory.

Certain countries in the expanded territory with Antengene became available following Karyopharm’s reacquisition of the exclusive development and commercial rights from Ono which were transferred to Karyopharm in April 2020 at no cost to Karyopharm. Karyopharm has chosen to retain the rights to selinexor and eltanexor in Japan while granting Antengene the exclusive rights to develop and commercialize selinexor and eltanexor in South Korea, Taiwan, Hong Kong and the ASEAN countries.

Outside of the territories licensed by Antengene, and the market for selinexor in Israel, Karyopharm maintains complete development and commercial rights to selinexor and eltanexor throughout the world, including in the U.S., Canada, Europe, Japan, and Latin America.
Strengthened the Balance Sheet with a Public Equity Offering. On March 6, 2020, Karyopharm completed an underwritten public offering of 7,187,500 shares of its common stock at a price to the public of $24.00 per share. The net proceeds of $161.8 million from the offering will be used to support key research and development and commercial initiatives, including potential future product launches.

Collaboration Agreement with Curadev Pharma Pvt Ltd (Curadev). Karyopharm and Curadev, a privately-owned biotechnology company, have entered into a strategic collaboration to identify and co-develop novel small molecules against various biological targets for the treatment of cancer and other major diseases. Under the terms of the agreement, Karyopharm and Curadev have agreed to identify and develop small molecules against up to two targets. Curadev will conduct exploratory research, drug discovery and development for designated programs up to the conclusion of preclinical proof of concept studies, after which Karyopharm will have an option to an exclusive license to develop and commercialize each target on a global basis. Karyopharm and Curadev will co-fund and jointly oversee development up to the option exercise period.

Key Appointments to the Executive Leadership Team and Board of Directors. During the first quarter of 2020, Karyopharm appointed John Demaree as Chief Commercial Officer and its Board of Directors elected Richard Paulson as a director. Mr. Demaree brings more than 20 years of oncology experience, building commercial capabilities and leading multiple successful product launches. Mr. Paulson currently serves as Executive Vice President and Chief Executive Officer of Ipsen North America and brings over 25 years of global biopharmaceutical industry experience, including launching best-in-class oncology medicines.
First Quarter 2020 Financial Results

Net product revenue: Net product revenue for the first quarter of 2020 was $16.1 million. Karyopharm commenced sales of XPOVIO in the U.S. during the third quarter of 2019 and therefore did not have net product revenue during the first quarter of 2019.

License and other revenue: License and other revenue for the first quarter of 2020 was $2.1 million, compared to $0.2 million for the first quarter of 2019. The increase was driven in part by the recognition of $1.1 million pursuant to our license agreement with Antengene.

Cost of sales: Cost of sales were $0.8 million for the first quarter of 2020. Cost of sales reflects the costs of XPOVIO units sold and third-party royalties on net product revenue.

Research and development expenses (R&D): R&D expenses for the first quarter of 2020 were $34.0 million, compared to $38.0 million for the first quarter of 2019.

Selling, general and administrative expenses (SG&A): For the first quarter of 2020, SG&A expenses were $30.7 million, compared to $27.1 million for the first quarter of 2019. The increase in SG&A expenses compared to the prior year was due primarily to activities to support the U.S. commercialization of XPOVIO and in preparation for the potential launch of additional indications in 2020.

Interest expense: Interest expense for the first quarter of 2020 was $6.5 million, compared to $3.0 million for the first quarter 2019. The increase in interest expense is attributable to the imputed interest on the deferred royalty obligation Karyopharm has with HealthCare Royalty Partners.

Net loss: Karyopharm reported a net loss of $52.9 million, or $0.78 per share, for the first quarter of 2020, compared to a net loss of $66.2 million, or $1.09 per share, for the first quarter of 2019. Net loss includes non-cash stock-based compensation expense of $5.2 million and $3.9 million for the 2020 and 2019 quarters, respectively.

Cash position: Cash, cash equivalents, restricted cash and investments as of March 31, 2020 totaled $385.2 million, compared to $265.8 million as of December 31, 2019.

2020 Financial Outlook

Karyopharm expects XPOVIO net product sales to be slightly higher in the second quarter of 2020 as compared to the first quarter of 2020. In addition, total revenues are expected to be higher due to an increase in collaboration revenue from the expanded territory agreement with Antengene. The Company will not be issuing XPOVIO revenue guidance for the full year 2020 as it continues to monitor the ongoing commercial impact from the COVID-19 pandemic as well as the timing of key expected regulatory actions in 2020. These regulatory events include the potential approval of XPOVIO for patients with relapsed or refractory DLBCL as well as Karyopharm’s planned sNDA submission, and subsequent FDA-review period, requesting expansion of the approved indication for XPOVIO to include second line treatment for patients with relapsed or refractory multiple myeloma.

Based on its current operating plans, including the reduction of some R&D costs as a result of trial delays due to the ongoing COVID-19 pandemic, Karyopharm expects its non-GAAP R&D and SG&A expenses, which excludes stock-based compensation expense, for the full year 2020 to be at the lower end of the previously projected range of $240 million to $260 million. This estimate includes the additional costs associated with our new selinexor clinical trial in patients with severe COVID-19. Karyopharm has not reconciled the full year 2020 outlook for non-GAAP R&D and SG&A expenses to full year 2020 outlook for GAAP R&D and SG&A expenses because Karyopharm cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the full year 2020 outlook for non-GAAP R&D and SG&A expenses.

The Company expects that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO product sales, will be sufficient to fund its planned operations into the middle of 2022.

Non-GAAP Financial Information

Karyopharm uses a non-GAAP financial measure, including R&D and SG&A expenses, to provide operating expense guidance. Non-GAAP R&D and SG&A expenses exclude stock-based compensation expense. Karyopharm believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Karyopharm’s operating performance as it excludes non-cash stock compensation expense. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP R&D and SG&A expenses and should not be considered a measure of Karyopharm’s liquidity. Instead, non-GAAP R&D and SG&A expenses should only be used to supplement an understanding of Karyopharm’s operating results as reported under GAAP.

Conference Call Information

Karyopharm will host a conference call today, Tuesday, May 5, 2020, at 8:30 a.m. Eastern Time, to discuss the first quarter 2020 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 9936655. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

IMPORTANT SAFETY INFORMATION

Thrombocytopenia

XPOVIO can cause thrombocytopenia, leading to potentially fatal hemorrhage. Thrombocytopenia was reported as an adverse reaction in 74% of patients, and severe (Grade 3-4) thrombocytopenia occurred in 61% of patients treated with XPOVIO. The median time to onset of the first event was 22 days. Bleeding occurred in 23% of patients with thrombocytopenia, clinically significant bleeding occurred in 5% of patients with thrombocytopenia and fatal hemorrhage occurred in <1% of patients.

Monitor platelet counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Institute platelet transfusion and/or other treatments as clinically indicated. Monitor patients for signs and symptoms of bleeding and evaluate promptly. Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse reaction.

Neutropenia

XPOVIO can cause neutropenia, potentially increasing the risk of infection. Neutropenia was reported as an adverse reaction in 34% of patients, and severe (Grade 3-4) neutropenia occurred in 21% of patients treated with XPOVIO. The median time to onset of the first event was 25 days. Febrile neutropenia was reported in 3% of patients.

Obtain neutrophil counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Monitor patients for signs and symptoms of concomitant infection and evaluate promptly. Consider supportive measures including antimicrobials for signs of infection and use of growth factors (e.g., G-CSF). Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse reaction.

Gastrointestinal Toxicity

Gastrointestinal toxicities occurred in patients treated with XPOVIO.

Nausea/Vomiting

Nausea was reported as an adverse reaction in 72% of patients, and Grade 3 nausea occurred in 9% of patients treated with XPOVIO. The median time to onset of the first nausea event was 3 days.

Vomiting was reported in 41% of patients, and Grade 3 vomiting occurred in 4% of patients treated with XPOVIO. The median time to onset of the first vomiting event was 5 days.

Provide prophylactic 5-HT3 antagonists and/or other anti-nausea agents, prior to and during treatment with XPOVIO. Manage nausea/vomiting by dose interruption, reduction, and/or discontinuation. Administer intravenous fluids and replace electrolytes to prevent dehydration in patients at risk. Use additional anti-nausea medications as clinically indicated.

Diarrhea

Diarrhea was reported as an adverse reaction in 44% of patients, and Grade 3 diarrhea occurred in 6% of patients treated with XPOVIO. The median time to onset of diarrhea was 15 days.

Manage diarrhea by dose modifications and/or standard anti-diarrheal agents; administer intravenous fluids to prevent dehydration in patients at risk.

Anorexia/Weight Loss

Anorexia was reported as an adverse reaction in 53% of patients, and Grade 3 anorexia occurred in 5% of patients treated with XPOVIO. The median time to onset of anorexia was 8 days.

Weight loss was reported as an adverse reaction in 47% of patients, and Grade 3 weight loss occurred in 1% of patients treated with XPOVIO. The median time to onset of weight loss was 15 days.

Monitor patient weight at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Manage anorexia and weight loss with dose modifications, appetite stimulants, and nutritional support.

Hyponatremia

XPOVIO can cause hyponatremia; 39% of patients treated with XPOVIO experienced hyponatremia, 22% of patients experienced Grade 3 or 4 hyponatremia. The median time to onset of the first event was 8 days.

Monitor sodium level at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first two months of treatment. Correct sodium levels for concurrent hyperglycemia (serum glucose >150 mg/dL) and high serum paraprotein levels. Treat hyponatremia per clinical guidelines (intravenous saline and/or salt tablets), including dietary review. Interrupt and/or reduce dose, or permanently discontinue based on severity of adverse reaction.

Infections

In patients receiving XPOVIO, 52% of patients experienced any grade of infection. Upper respiratory tract infection of any grade occurred in 21%, pneumonia in 13%, and sepsis in 6% of patients. Grade ≥3 infections were reported in 25% of patients, and deaths resulting from an infection occurred in 4% of patients. The most commonly reported Grade ≥3 infections were pneumonia in 9% of patients, followed by sepsis in 6%. The median time to onset was 54 days for pneumonia and 42 days for sepsis. Most infections were not associated with neutropenia and were caused by non-opportunistic organisms.

Neurological Toxicity

Neurological toxicities occurred in patients treated with XPOVIO.

Neurological adverse reactions including dizziness, syncope, depressed level of consciousness, and mental status changes (including delirium and confusional state) occurred in 30% of patients, and severe events (Grade 3-4) occurred in 9% of patients treated with XPOVIO. Median time to the first event was 15 days.

Optimize hydration status, hemoglobin level, and concomitant medications to avoid exacerbating dizziness or mental status changes.

Embryo-Fetal Toxicity

Based on data from animal studies and its mechanism of action, XPOVIO can cause fetal harm when administered to a pregnant woman. Selinexor administration to pregnant animals during organogenesis resulted in structural abnormalities and alterations to growth at exposures below those occurring clinically at the recommended dose.

Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with a female partner of reproductive potential to use effective contraception during treatment with XPOVIO and for 1 week after the last dose.

ADVERSE REACTIONS

The most common adverse reactions (incidence ≥20%) are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea, and upper respiratory tract infection.

The treatment discontinuation rate due to adverse reactions was 27%; 53% of patients had a reduction in the XPOVIO dose, and 65.3% had the dose of XPOVIO interrupted. The most frequent adverse reactions requiring permanent discontinuation in 4% or greater of patients who received XPOVIO included fatigue, nausea, and thrombocytopenia. The rate of fatal adverse reactions was 8.9%.

Bio-Techne To Present At The BofA Securities 2020 Health Care Conference

On May 5, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported that Chuck Kummeth, President and Chief Executive Officer, will present at the virtual BofA Securities 2020 Health Care Conference on Wednesday, May 13, 2020 at 9:40 a.m. EDT (Press release, Bio-Techne, MAY 5, 2020, View Sourcenews/detail/188/bio-techne-to-present-at-the-bofa-securities-2020-health-care-conference" target="_blank" title="View Sourcenews/detail/188/bio-techne-to-present-at-the-bofa-securities-2020-health-care-conference" rel="nofollow">View Source [SID1234556997]). A live webcast of the presentation can be accessed via Bio-Techne’s Investor Relations website at View Source or through the following link http://www.veracast.com/webcasts/bofa/healthcare2020/id62109638.cfm.

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