On August 5, 2022 Oncolys BioPharma reported its non-consolidated financial results for the Six months ended June 30, 2022 (Press release, Oncolys BioPharma, AUG 5, 2022, View Source [SID1234618958]).

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On August 5, 2022 Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), a biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders, reported financial results for the second quarter ended June 30, 2022, and provided a review of recent accomplishments during and anticipated upcoming milestones (Press release, Biohaven Pharmaceutical, AUG 5, 2022, View Source [SID1234617686]).

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "As expected, we observed a strong rebound in NURTEC ODT net revenue as first quarter seasonal dynamics abated. Net product revenue of $194 million for the quarter represented a 57% sequential increase compared to the first quarter and reflects strong product demand. In addition, the sales mix of our 2-pack (16-tablet count) of Nurtec ODT continued to ramp in the second quarter. Investments made in first quarter copay programs drove volume and net revenue growth, and the benefits of the only ‘all-in-one’ therapy to treat and prevent migraine continued to resonate with and improve the lives of patients living with migraine. And now, with the intranasal zavegepant NDA filing acceptance, we are closer to bringing a new, critical medication to market that offers migraine patients ultra-rapid relief in as early as 15 minutes, and addresses the needs of patients experiencing nausea or vomiting who need non-oral treatment options."

Dr. Coric continued, "We are excited about the vast opportunities beyond CGRP and have made tremendous strides across our broader pipeline, starting with our program in focal epilepsy with what we believe is a best-in-class approach to Kv7 potassium channel activation. We are working hard to expedite enrollment in our Phase 1 trial and look forward to exploring its potential in other epilepsy types as well as in pain and affective disorders. Likewise, we recently dosed the first subject in our Phase 3 trial evaluating taldefgrobep alfa in subjects with Spinal Muscular Atrophy (SMA); we look forward to driving enrollment in this trial and exploring the potential of taldefgrobep alfa in additional indications. We also remain on track to report topline results from the verdiperstat arm of the platform trial in ALS, sponsored by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in the second half of the year. We are excited about the paradigm-shifting approaches we’re exploring across our expanding pipeline and will continue working determinedly to improve outcomes for patients living with neuroscience diseases."

Second Quarter and Recent Business Highlights

Q2 2022 net product revenue from sales of NURTEC ODT totaled $194 million, up sequentially 57% compared to Q1 2022 – NURTEC ODT has now achieved over 2,200,000 prescriptions, and over 79,000 unique prescribers through June 30, 2022, an increase of 11,500 prescribers from the first quarter, signaling continued traction across the prescribing community. The $194 million in net product revenues for the quarter represents a 109% increase over the second quarter of 2021 and a 57% increase from net product revenues over the first quarter of 2022. The increase of $70 million in net product revenues as compared to the first quarter of 2022 is due primarily to increased NURTEC ODT prescription sales volume and favorable pack mix per prescription. In addition, we continue to see improvements in patient affordability / copay support needs as patients satisfy annual deductible obligations. These are marginally offset by additional rebates during the second quarter of 2022 compared to the first quarter of 2022. The Company remains on track to meet full year 2022 net product sales guidance of $825 – $900 million.
Antitrust approval granted for proposed acquisition by Pfizer; other closing conditions – As previously disclosed, Biohaven entered into an agreement and plan of merger with Pfizer, Inc. ("Pfizer") on May 10, 2022, under which Pfizer will acquire Biohaven (the "Merger"). Under the terms of the agreement, Pfizer will acquire all outstanding shares of Biohaven not already owned by Pfizer for $148.50 per share in cash. Biohaven common shareholders, including Pfizer, will also receive 0.5 of a share of New Biohaven, a new publicly traded company that will retain Biohaven’s non-CGRP development stage pipeline compounds, per Biohaven common share. The boards of directors of both Biohaven and Pfizer have unanimously approved the transaction. Pfizer will pay transaction consideration totaling approximately $11.6 billion in cash. Pfizer will also make payments at closing to settle Biohaven’s third party debt and for the redemption of all outstanding shares of Biohaven’s redeemable preferred stock.
Consummation of the Merger is conditioned upon, among other things, expiration or termination of any applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended (the "HSR Act"). The applicable waiting period under the HSR Act expired at 11:59 p.m., ET, on June 27, 2022.
In addition to antitrust approval in the United States, the Merger was cleared by the German Federal Cartel Office on June 13, 2022, and was cleared by the Austrian Federal Competition Authority on June 23, 2022. On June 24, 2022, in response to a briefing paper filed by Pfizer on May 20, 2022, the UK Competition and Markets Authority confirmed that no further information is required on the Merger.
The Merger transaction is also subject to completion of the New Biohaven spin-off transaction and customary closing conditions, including the approval of Biohaven’s shareholders. The Company filed a preliminary proxy statement with the United States Securities and Exchange Commission ("SEC") on July 1, 2022.
U.S. FDA accepted Biohaven’s zavegepant NDA filing for the acute treatment of migraine in adults – In May, the Company announced that the FDA filed and accepted for review the Company’s recently submitted NDA for zavegepant, the only small molecule calcitonin gene-related peptide ("CGRP") receptor antagonist in an intranasal formulation, for the acute treatment of migraine in adults. The Prescription Drug User Fee Act ("PDUFA") goal date for completion of the FDA review of the NDA is set for the first quarter of 2023. The Company had announced the achievement of positive results in the pivotal trial of zavegepant for the acute treatment of migraine in adults in December, 2021.
Commenced enrollment in Phase 3 Spinal Muscular Atrophy ("SMA") study – In July, the Company commenced enrollment in a Phase 3 clinical trial assessing the efficacy and safety of taldefgrobep alfa in Spinal Muscular Atrophy (SMA). Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth, through two mechanisms: lowering myostatin directly and blocking key downstream signaling mechanisms. The Company expects to enroll approximately 180 patients in this randomized, double-blind, placebo-controlled global trial.
Global Coalition for Adaptive Research ("GCAR") commenced enrollment in Glioblastoma Adaptive Global Innovative Learning Environment ("GBM Agile") Phase 2-3 adaptive platform trial for patients with glioblastoma – In July, GCAR announced the activation of Biohaven’s troriluzole and Vigeo Therapeutics’ VT1021 in GBM AGILE, a patient-centered, adaptive platform trial for registration that tests multiple therapies for patients with newly-diagnosed and recurrent glioblastoma ("GBM"). GBM AGILE is an international, innovative platform trial designed to more rapidly identify and confirm effective therapies for patients with glioblastoma through response adaptive randomization. The new interventions are opening first at Henry Ford Health Cancer in Detroit under Henry Ford site Principal Investigator Dr. Tom Mikkelsen and will subsequently open at more than 40 trial sites across the United States with additional global sites to follow.
Phase 3 clinical trial of troriluzole did not reach statistical significance in overall SCA population – In May, the Company announced top-line results from a Phase 3 clinical trial evaluating the efficacy and safety of its investigational therapy, troriluzole, in patients with spinocerebellar ataxia (SCA). The primary endpoint, change from baseline to Week 48 on the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA), did not reach statistical significance in the overall SCA population as there was less than expected disease progression over the course of the study. However, post hoc analysis of efficacy measures by genotype suggested a treatment effect in patients with the SCA Type 3 (SCA3) genotype, which represents the most common form of SCA and accounted for 41 percent of the study population. In the SCA3 subgroup, troriluzole showed a numerical treatment benefit on the change in f-SARA score from baseline to Week 48 compared to placebo. Given the debilitating nature of the disease and lack of approved therapies, the Company intends to share the SCA3 genotype data with regulators and work with the FDA to address a path forward for troriluzole in SCA.
Presented new Nurtec ODT, zavegepant, and migraine health economics and outcomes research (HEOR) data at the American Headache Society Annual Scientific Meeting – In June, the Company presented 31 new and encore presentations, including three late-breakers and three oral presentations, at the 64th annual scientific meeting of the American Headache Society. Key updates:
The Company presented full Phase 3 results for zavegepant nasal spray as an acute treatment of migraine as well as data from a 52-week open label extension study of Nurtec ODT (rimegepant) evaluating every other day preventive treatment of migraine plus as an as-needed acute treatment, and estimated reductions in monthly migraine days (MMDs) with rimegepant acute treatment.
Late-breaking submissions highlighted new data about patterns of medication utilization and migraine frequency for adults using Nurtec ODT as a preventive and acute treatment, results from a Phase 1 trial of Nurtec ODT in healthy Chinese adults, and outcomes of a Phase 3 study of Nurtec ODT conducted in China and Korea as an acute treatment of migraine.
Several HEOR posters notably highlighted (1) reductions in the prevalence of medication overuse headache following Nurtec ODT initiation, (2) reduced mean monthly quantity of dispensed Nurtec ODT which is potentially reflective of MMD reductions, (3) patient survey studies that illustrate preference for ODT oral formulations versus injectable or IV treatments in prevention, (4) improvements in health-related quality of life with long-term Nurtec ODT treatment, (5) the high interictal burden of migraine, and (6) patient reported attenuation of effect with biologic treatment in prevention.
Upcoming Milestones:

Biohaven is continuing to support the commercialization of NURTEC ODT for the acute and preventive treatment of migraine, as well as develop its product candidates through clinical and preclinical programs in a number of common and rare disorders. The Company expects to reach significant pipeline milestones across its platforms in the coming quarters.

Biohaven expects to:

Consummate the acquisition by Pfizer by early 2023, subject to the completion of the new Biohaven spin-off transaction and other customary closing conditions.
Advance Phase 1 study of BHV-7000 (Kv7) in focal epilepsy.
Continue to advance the commercialization of NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine.
Continue to advance the zavegepant (intranasal spray) program towards commercialization for the acute treatment of migraine.
Submit an NDA for the acute treatment of migraine in China in the second half of 2022.
Report topline of verdiperstat in ALS in the second half of 2022.
Complete enrollment in Phase 3 study of troriluzole in Obsessive-Compulsive Disorder in 2023.
Capital Position:

Cash, cash equivalents, and marketable securities as of June 30, 2022, were $553.5 million, excluding $0.8 million of restricted cash, compared to $364.6 million as of December 31, 2021. On January 4, 2022, we received $500.0 million in upfront proceeds from Pfizer relating to our strategic collaboration arrangement, consisting of $150.0 million cash and $350.0 million in proceeds from the purchase of Biohaven common shares at a 25% market premium. In addition, during the second quarter of 2022 we drew $125.0 million in non-dilutive committed capital from our credit facility with Sixth Street.

Second Quarter 2022 Financial Highlights

Product Revenue, Net: Net product revenue was $194.0 million for the three months ended June 30, 2022, compared to $92.9 million for the three months ended June 30, 2021. The increase of $101.0 million in net product revenue was primarily due to increased NURTEC ODT sales volume as well as an increase in average pills per prescription during the three months ended June 30, 2022, compared to the three months ended June 30, 2021. The increase in the second quarter of 2022 compared to the second quarter of 2021 was also benefited from improved patient affordability support, partially offset by additional rebate related sales allowances.

Collaboration and Other Revenue: Collaboration and other revenue was $21.1 million for the three months ended June 30, 2022. No collaboration and other revenue was recognized for the three months ended June 30, 2021. The collaboration and other revenue recognized during the three months ended June 30, 2022 was primarily due to $20.0 million of variable consideration recognized as part of our collaboration arrangement with Pfizer.

Research and Development ("R&D") Expenses: R&D expenses, including non-cash share-based compensation costs, were $218.5 million for the three months ended June 30, 2022, compared to $77.4 million for the three months ended June 30, 2021. The increase of $141.1 million was primarily due to the acquisition of our Kv7 Platform from Knopp Biosciences LLC in April 2022 for $93.7 million and a $25.0 million development milestone related to BHV-7000, increased program expenses for rimegepant, and increased personnel costs. Non-cash share-based compensation expense was $16.6 million for the three months ended June 30, 2022, an increase of $7.4 million as compared to the same period in 2021.

Selling, General and Administrative ("SG&A") Expenses: SG&A expenses, including non-cash share-based compensation costs, were $250.5 million for the three months ended June 30, 2022, compared to $170.1 million for the three months ended June 30, 2021. The increase of $80.4 million was primarily due to increases in spending to support increased commercial sales of NURTEC ODT, including the launch of NURTEC ODT for the preventative treatment of migraine which was approved by the FDA in May of 2021. Additionally, approximately $9.6 million of the increased SG&A expense related to fees incurred in connection with the Pfizer Merger, including increased legal and accounting costs. Less than half of the SG&A expense was for commercial organization personnel costs, excluding non-cash share-based compensation expense. Non-cash share-based compensation expense was $23.6 million for the three months ended June 30, 2022, an increase of $7.3 million as compared to the same period in 2021.

Other Expense, Net: Other expense, net was $152.2 million for the three months ended June 30, 2022, compared to $34.9 million for the three months ended June 30, 2021. The increase of $117.3 million in net expense was primarily due to a $109.7 million change in fair value of derivative liabilities, as well as $9.3 million of increased interest expense as a result of additional borrowings under our debt facility with Sixth Street Specialty Lending, Inc. ("Sixth Street"). The change in fair value of derivative liabilities was a result of derivative liabilities recorded in connection with change of control provisions associated with the pending acquisition of Biohaven by Pfizer for our series A and B preferred shares and term loans with Sixth Street.

Net Loss: Biohaven reported a net loss attributable to common shareholders for the three months ended June 30, 2022, of $441.4 million, or $6.21 per share, compared to $210.6 million, or $3.23 per share for the same period in 2021. Anticipated closing of the merger agreement necessitates accounting of derivative losses in the amount of $1.57 per share resulting in a total second quarter reported net loss of $6.21 per share. Non-GAAP adjusted net loss for the three months ended June 30, 2022 was $271.4 million, or $3.82 per share, compared to $170.9 million, or $2.62 per share for the same period in 2021. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges, non-cash interest expense related to the accounting for mandatorily redeemable preferred shares and liability related to sale of future royalties, changes in the fair value of derivatives and gains or losses from equity method investment. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain non-GAAP financial measures. In particular, Biohaven has provided non-GAAP adjusted net loss and adjusted net loss per share, adjusted to exclude the items below. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, Biohaven believes the presentation of non-GAAP adjusted net loss and adjusted net loss per share, when viewed in conjunction with GAAP results, provides investors with a more meaningful understanding of ongoing operating performance. These measures exclude (i) non-cash share-based compensation, which is substantially dependent on changes in the market price of common shares, (ii) interest expense related to the accounting for our mandatorily redeemable preferred shares and liability related to sale of future royalties, which are in excess of the actual interest owed, (iii) changes in the fair value of derivative liability, which does not correlate to actual cash payment obligations in the relevant periods, and (iv) gains or losses from equity method investment, which are non-cash and based on the financial results and valuation of another company that we did not manage or control.

Biohaven believes the presentation of these non-GAAP financial measures provides useful information to management and investors regarding Biohaven’s results of operations. When GAAP financial measures are viewed in conjunction with these non-GAAP financial measures, investors are provided with a more meaningful understanding of Biohaven’s ongoing operating performance and are better able to compare Biohaven’s performance between periods. In addition, these non-GAAP financial measures are among those indicators Biohaven uses as a basis for evaluating performance, and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this press release.

On August 4, 2022 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported business highlights and financial results for the quarter ended June 30, 2022 (Press release, Karyopharm, AUG 4, 2022, View Source [SID1234617454]).

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"I’m pleased with our team’s ongoing commitment to successfully execute against key priorities in the second quarter, achieving more than 75% year-over-year revenue growth and further expanding patient access for selinexor globally following full marketing authorization from the European Commission and recent launches in China and Canada," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "Despite headwinds caused by COVID-19 at the beginning of the year and increased competition for later lines of multiple myeloma treatment, we continue to see increased use of XPOVIO in earlier lines with growth in the community setting. Looking ahead to the remainder of the year, we have several key upcoming milestones, including reporting additional data from our studies of selinexor in front-line myelofibrosis and eltanexor in relapsing/refractory myelodysplastic syndromes."

Second Quarter 2022 and Recent Highlights

XPOVIO Commercial Performance

Achieved U.S. net product revenue for the second quarter of 2022 of $29.0 million, a 44% increase compared to the second quarter of 2021, driven by strong year-over-year growth in new patient starts and refills, with continued shift of XPOVIO into second to fourth lines of therapy.

In the second quarter of 2022, a recovery in new patient starts as compared to the previous quarter was offset by the decline in refills due to COVID-19 related reduction of new patient starts in the beginning of the year and intensified late-line competition in the academic setting.

XPOVIO continued its growth in the community setting, driven by a positive shift in perception and intent to treat metrics.

Increased selinexor’s reach to patients around the world with the full marketing authorization of NEXPOVIO (selinexor) in Europe, which expands the indication in multiple myeloma to patients with multiple myeloma after at least one prior therapy as well as commercial launches in mainland China and Canada by partners Antengene Corporation Limited and FORUS Therapeutics Inc., respectively.
Research & Development (R&D) Highlights for Selinexor and Eltanexor

European Commission granted marketing authorization of NEXPOVIO in combination with once weekly bortezomib (Velcade) and low-dose dexamethasone (XVd) for the treatment of adults with multiple myeloma who have received at least one prior therapy, expanding on the prior approval which was in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

Results from Phase 1/2 study of selinexor in combination with ruxolitinib in treatment-naïve myelofibrosis were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2022 Annual Meeting, including a generally manageable side effect profile with no dose limiting toxicities and 75% of evaluable patients demonstrating ≥35% spleen volume reduction (SVR 35) at week 12. The most commonly reported Grade 3/4 treatment emergent adverse events were thrombocytopenia (27%), anemia (20%) and neutropenia (20%). These data were also presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress.

Following productive discussions with the U.S. Food and Drug Administration (FDA), the Company is finalizing a partner for a companion diagnostic to be used in a registration-enabling study of selinexor in patients with p53 wild-type endometrial cancer.

FDA granted fast track designation for the development program of eltanexor as monotherapy for the treatment of patients with relapsed or refractory intermediate, high-, or very high-risk myelodysplastic syndromes (MDS), per IPSS-R. In addition, the FDA also granted eltanexor orphan drug designation for the treatment of MDS and selinexor for the treatment of myelofibrosis.

The European Commission granted EU orphan medicinal product designation for eltanexor for the treatment of MDS.
2022 Financial Guidance

Based on its current operating plans, Karyopharm is updating its guidance for full year 2022:

Total revenue to be in the range of $155 million to $165 million.

XPOVIO net product revenue to be in the range of $120 million to $130 million versus previous guidance of $135 million to $145 million.

Non-GAAP R&D and Selling, general and administrative (SG&A) expenses, excluding stock-based compensation expense, to be in the range of $250 million to $265 million versus previous guidance of $265 million to $280 million.

Karyopharm has not reconciled the full year 2022 outlook for non-GAAP R&D and SG&A expenses to full year 2022 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 2022 outlook for non-GAAP R&D and SG&A expenses.

The Company continues to expect that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO product sales, as well as revenue generated from its license agreements, will be sufficient to fund its planned operations into early 2024.
Second Quarter 2022 Financial Results

Total Revenues: Total revenue for the second quarter of 2022 was $39.7 million, up 76% compared to $22.6 million for the second quarter of 2021.

Net product revenue: Net product revenue for the second quarter of 2022 was $29.0 million, up 44% compared to $20.2 million for the second quarter of 2021.

License and other revenue: License and other revenue for the second quarter of 2022 was $10.7 million, compared to $2.4 million for the second quarter of 2021. The increase was primarily attributable to $6.5 million earned in reimbursement of development expenses from the Menarini Group.

Cost of sales: Cost of sales for the second quarter of 2022 were $0.9 million, compared to $1.1 million for the second quarter of 2021. Cost of sales reflects the costs of XPOVIO units sold and third-party royalties on net product revenue.

R&D expenses: R&D expenses for the second quarter of 2022 were $44.3 million, compared to $34.0 million for the second quarter of 2021. The increase was primarily driven by an increase in personnel costs and stock-based compensation, including $3.8 million of severance-related stock-based compensation expense incurred during the three months ended June 30, 2022.

SG&A expenses: SG&A expenses for the second quarter of 2022 were $37.3 million, compared to $36.5 million for the second quarter of 2021. The increase in SG&A expenses was primarily due to an increase in stock-based compensation as a result of $3.5 million of severance-related stock-based compensation expense incurred during the three months ended June 30, 2022.

Interest expense: Interest expense for the second quarter of 2022 was $6.3 million, compared to $5.0 million for the second quarter of 2021.

Net loss: Karyopharm reported a net loss of $49.1 million, or $0.62 per share, for the second quarter of 2022, compared to a net loss of $53.6 million, or $0.71 per share, for the second quarter of 2021. Net loss included non-cash stock-based compensation expense of $15.1 million and $8.1 million for the second quarters of 2022 and 2021, respectively.

Cash position: Cash, cash equivalents, restricted cash and investments as of June 30, 2022, totaled $172.6 million, compared to $235.6 million as of December 31, 2021.

Non-GAAP Financial Information

Karyopharm uses a non-GAAP financial measure, non-GAAP 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, August 4, 2022, at 8:00 a.m. Eastern Time, to discuss the second quarter 2022 financial results and provide other business highlights. To access the conference call, please dial (888) 349-0102 (local) or (412) 902-4299 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call, along with accompanying slides, 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.

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, China, South Korea, Canada and Israel, and is marketed in those areas by Karyopharm’s global partners.

Please refer to the local Prescribing Information for full details.

Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.

The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations
Lactation: Advise not to breastfeed.

On August 4, 2022 Purple Biotech Ltd. ("Purple Biotech", or the "Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class, effective and durable therapies by harnessing the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, reported financial results for the second quarter ended June 30, 2022 (Press release, Purple Biotech, AUG 4, 2022, View Source [SID1234617475]).

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"This is the first report of Purple Biotech financial results since my appointment as Chief Executive Officer," said Gil Efron, Chief Executive Officer of Purple Biotech. "It is an opportunity for me to emphasize our main objectives. Purple Biotech has two exciting and innovative lead assets in development. We are focused on performing robust studies aiming to achieve meaningful clinical data and are continuing to explore opportunities to expand our current clinical programs to additional indications, while maintaining our cash runway, currently through the end of 2024. We are seeking opportunities for additional assets and collaborations to increase our footprint, through accretive transactions. I believe that together with strong science and by leveraging on our capabilities we can achieve these objectives and solidify Purple Biotech as a significant player within the oncology field."

Recent Corporate Highlights

NT219

In June, Purple presented Phase 1 interim monotherapy data of the ongoing Phase 1/2 clinical trial of NT219 at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, demonstrating encouraging safety & efficacy profile including one confirmed partial response in gastroesophageal junction cancer patient and stable disease in 75% of patients with mutated-KRAS colorectal cancer.

CM24

The Company initiated the Phase 2 portion of its ongoing study of CM24, a first-in-class monoclonal antibody with the potential to treat multiple cancers. The Phase 2 is an open-label, multicenter study in patients with metastatic pancreatic cancer (PDAC) to evaluate the safety and tolerability of CM24 in combination with the PD-1 inhibitor Opdivo (nivolumab) and chemotherapy. The primary study endpoint is to evaluate preliminary efficacy in 2nd line PDAC.

This follows the recent favorable safety and efficacy data supporting the advancement of CM24 at the recommended phase 2 dose of 20 mg/Kg. The data from the Phase 1b study of CM24 in combination with Opdivo (nivolumab) was presented in a poster entitled "Interim Safety and Efficacy Results from a Phase 1b Study of CM24 in Combination with Nivolumab in Adults with Advanced Solid Tumors" at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting in May.

"Based on this progress last quarter, starting from phase 2, we will be performing randomized studies, such as our CM24 study in PDAC, which we are now in the process of expanding accordingly," added Efron. "The recent clinical data for NT219 together with previously published preclinical results have shown the potential of NT219 on solid tumors harboring KRAS mutation and we are looking into the development of NT219 for treatment of mutated KRAS patients. In addition, Insulin Receptor Substrates 1/2 (IRS) was identified as one of the pathways for resistance mechanism to other treatments in this field, opening this opportunity for NT219, which is the only IRS inhibitor in clinical development to date."

"We are ending the quarter in a strong financial position with $38.7 million in cash, cash equivalent short term and long term deposits and a cash runway of two and a half years as we continue to control our costs while advancing our programs. I’m proud of the progress made in the last quarter and we look forward to continuing to advance our clinical stage programs on behalf of cancer patients."

Financial Results for the three Months Ended June 30, 2022

Research and Development Expenses were $2 million, same as to $2 million in the same period of 2021.

Selling, General and Administrative Expenses were $1.5 million, same as to $1.5 million in the same period of 2021.

Operating Loss was $3.6 million, same as the $3.6 million in the same period of 2021.

On a non-IFRS basis (as reconciled below), adjusted operating loss was $3 million, an increase of $0.1 million, compared to $2.9 million in the same period of 2021.

Net Loss for the three months ended June 30, 2022 was $3.6 million, or $0.20 per basic and diluted ADS, compared to a net loss of $3.6 million, or $0.21 per basic and diluted ADS, in the three months ended June 30, 2021. Adjusted net loss for the three months ended June 30, 2022 was $2.9 million, the same as in the three months ended June 30, 2021.

Financial Results for the Six Months Ended June 30, 2022

Research and Development Expenses were $8.0 million, an increase of $1.1 million, or 15.9%, compared to $6.9 million in the same period of 2021. The increase was mainly due to an increase of $500 thousand in CMC expenses in support of our clinical studies and $300 thousands in wages in support of our growing development activities.

Sales, General and Administrative Expenses were $2.9 million, compared to $3.1 million in the same period of 2021, a decrease of $0.2 million. The decrease was mainly due to a decrease in employee equity-based compensation (ESOP) costs.

Operating Loss from continuing operations was $10.9 million, an increase of $0.8 million, or 7.9%, compared to $10.1 million in the same period of 2021.

On a non-IFRS basis (as reconciled below), adjusted operating loss was $10 million, an increase of $1.3 million, compared to $8.7 million in the same period of 2021, mainly due to an increase in R&D expenses.

Net Loss for the first six months ended June 30, 2022 was $10.8 million, or $0.61 per basic and diluted ADS, compared to a net loss of $10.2 million, or $0.58 per basic and diluted ADS, in the same period of 2021. The increase in net loss was mainly due to an increase of $0.8 million in operating expenses. Adjusted net loss for the first six months ended June 30, 2022 was $10 million, an increase from $8.7 million in the first six months ended June 30, 2021.

During the six months ended June 30, 2022, the Company sold, under the Open Market Sale Agreementsm with Jefferies LLC, approximately 179 thousand ADSs, at an average price of $3.64 per ADS. Net proceeds to the Company, were approximately $0.57 million, net of issuance expenses.

On August 4, 2022 The Bayer Group reported that achieved substantial growth in the second quarter of 2022 (Press release, Bayer, AUG 4, 2022, View Source [SID1234617523]). "We delivered strong operational performance. In terms of sales, we posted significant gains at Crop Science and strong growth at Consumer Health, as well as a slight increase at Pharmaceuticals, too. And with EBITDA before special items, we even achieved growth of 30 percent," said Werner Baumann, Chairman of the Board of Management, on Thursday while presenting the company’s half-year financial report. "In view of our good business performance and higher growth expectations, we have raised our full-year guidance," he explained.

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Bayer does not currently see any material financial impact in 2022 from any potential gas supply bottlenecks as a result of the war in Ukraine, Baumann said. The company has taken steps to ensure that the direct impact of any potential gas shortages on its own production capabilities this year is contained to the greatest degree possible, he explained. "From a technical perspective, we are well prepared to significantly reduce our reliance on natural gas by switching to alternative and renewable sources of energy. We have also launched programs to save energy and have built up our stocks of products where possible." A higher degree of uncertainty stems from the company’s indirect exposure via its global supplier network, Baumann said. "That’s why we are expanding our network of suppliers and building up additional inventory of key raw materials and packaging materials."

In the second quarter of 2022, Group sales increased by 9.6 percent to 12.819 billion euros on a currency- and portfolio-adjusted basis (Fx & portfolio adj.). EBITDA before special items advanced by 30.0 percent to 3.349 billion euros. Positive currency effects benefited sales by 915 million euros (Q2 2021: minus 524 million euros) and EBITDA before special items by 300 million euros (Q2 2021: minus 153 million euros). EBIT came in at 169 million euros (Q2 2021: minus 2.281 billion euros) after net special charges of 2.111 billion euros (Q2 2021: 3.901 billion euros) that mainly comprised 1.322 billion euros in impairment losses on intangible assets at the Crop Science Division. These impairment losses were recognized as part of impairment testing that was performed due to a strong rise in capital market interest rates. Other special charges related to ongoing litigations and restructuring measures.

Bayer has taken an additional provision of 694 million euros in the second quarter mainly due to ongoing settlement negotiations with the State of Oregon. The settlement, when finalized, would resolve a pending environmental impairment case, involving legacy Monsanto PCB products, and result in the dismissal of the case. Given the unique challenges, trial procedures, and substantive law for this case in this Oregon venue, the company decided to pursue settlement, even though Monsanto voluntarily ceased production of PCBs in 1977 and never disposed of the chemical in this state. Bayer remains committed to defend future cases through the litigation process. Recently, a Delaware State Court dismissed all of the state’s claims in a similar PCB-related case alleging environmental impairments.

Monsanto has broad indemnity agreements with its former customers and the company will pursue its rights to recover costs associated with the PCB-related litigation. The company recently filed a complaint in the Circuit Court of St. Louis County for the State of Missouri to enforce its rights.

In the second quarter, net income amounted to minus 298 million euros (Q2 2021: minus 2.335 billion euros), while core earnings per share from continuing operations rose by 19.9 percent to 1.93 euros.

Free cash flow was level with the prior-year period, at 1.140 billion euros. Net financial debt as of June 30, 2022, came in at 36.575 billion euros, up 5.9 percent from March 31, 2022.

Significantly improved market environment at Crop Science

In the agricultural business (Crop Science), Bayer increased sales by 17.2 percent (Fx & portfolio adj.) to 6.461 billion euros, driven by a substantial improvement in the market environment. The division recorded double-digit percentage growth in Latin America and Europe/Middle East/Africa, and also registered an expansion of business in North America and Asia/Pacific. Herbicides posted the strongest growth, at 51.3 percent (Fx & portfolio adj.), with sales rising particularly in Latin and North America, as well as in Europe/Middle East/Africa as a result of prices for glyphosate-based products remaining high. Sales at Corn Seed & Traits advanced by 9.5 percent (Fx & portfolio adj.), mainly due to price increases in North America, Europe/Middle East/Africa and Latin America. In addition, volumes expanded in all regions except North America. Sales at Fungicides rose by 4.3 percent (Fx & portfolio adj.), with growth in all regions except North America, where volumes declined as a result of unfavorable weather conditions. Soybean Seed & Traits saw sales decline by 16.1 percent (Fx & portfolio adj.), largely due to the significant reduction in sales from overproduction in North America and the unit’s withdrawal from the Argentinian market.

EBITDA before special items at Crop Science climbed by 71.8 percent to 1.749 billion euros. The growth in earnings was mainly driven by the substantial improvement in business performance, as well as contributions from ongoing efficiency programs. There was also a positive currency effect of 215 million euros (Q2 2021: minus 111 million euros). By contrast, earnings were diminished by an increase in costs, particularly in the cost of goods sold, which was mainly due to high inflation. The EBITDA margin before special items rose by 6.8 percentage points to 27.1 percent.

Successful product launches at Pharmaceuticals

Sales of prescription medicines (Pharmaceuticals) increased by 2.1 percent (Fx & portfolio adj.) to 4.818 billion euros. The division’s new products, especially Nubeqa and Kerendia, continued their successful market launch, with sales of the cancer drug Nubeqa doubling compared with the prior-year quarter. However, overall sales growth was held back by additional tender procedures in China, particularly for the cancer drug Nexavar and the oral anticoagulant Xarelto, which saw their global sales fall by 29.5 percent (Fx & portfolio adj.) and 6.4 percent (Fx & portfolio adj.), respectively. Xarelto sales were also impacted by the expiration of the product’s patent in Brazil. By contrast, sales of the ophthalmology drug Eylea rose by 11.7 percent (Fx & portfolio adj.), with business up in all regions. The company was able to capture market share, particularly in Europe, thanks in part to Eylea prefilled syringes. The division also registered significant growth for Adalat, its heart disease treatment, and Aspirin Cardio, its product for secondary prevention of heart attacks. Sales of these two products were up 11.2 percent (Fx & portfolio adj.) and 18.9 percent (Fx & portfolio adj.), respectively, due to higher volumes in China. Sales of the cancer drug Stivarga increased at an even stronger rate of 27.6 percent (Fx & portfolio adj.), mainly driven by expanded volumes in China and the United States.

EBITDA before special items at Pharmaceuticals advanced by 4.9 percent to 1.478 billion euros. Higher raw material costs and increased marketing investments in new products were largely offset by the growth in sales. The division also generated income from the sale of non-core businesses. There were positive currency effects of 41 million euros (Q2 2021: minus 26 million euros). The EBITDA margin before special items amounted to 30.7 percent.

Consumer Health grows business in all regions

Bayer’s sales of self-care products (Consumer Health) advanced by 6.8 percent (Fx & portfolio adj.) to 1.496 billion euros, with broad-based growth in all regions and nearly all categories. Sales in the Allergy & Cold category rose by 16.9 percent (Fx & portfolio adj.), largely due to continuously high cold incidence rates in Europe and North America. In June, the division also started selling Astepro, a product that it switched from Rx to OTC status. As the first and only steroid-free antihistamine nasal spray available over the counter on the U.S. market, Astepro is a differentiated, fast-working solution. Sales rose by a double-digit percentage (Fx & portfolio adj.) in the Digestive Health category as well, and the Dermatology and Pain & Cardio categories also recorded significant growth. After posting substantial gains since 2020, sales in the Nutritionals category declined by 3.7 percent (Fx & portfolio adj.) but remained at a high level overall.

EBITDA before special items at Consumer Health climbed by 18.7 percent to 330 million euros. The growth in earnings was on the back of a strong rise in sales, as well as the division’s continuous cost and price management efforts in an environment of accelerating inflation. There were also positive currency effects of 49 million euros (Q2 2021: minus 20 million euros). The EBITDA margin before special items rose by 0.5 percentage points to 22.1 percent.

Outlook for 2022 raised

Following the positive development of business in the first half of 2022, Bayer remains optimistic for the remainder of the year. It has therefore raised its guidance for the Crop Science and Consumer Health divisions, and thus also for the Group as a whole.

On a currency-adjusted basis (i.e. based on the average monthly exchange rates from 2021), Bayer now expects to generate sales of 47 billion to 48 billion euros in 2022 (previously: approximately 46 billion euros). This now corresponds to an increase of about 8 percent (previously: around 5 percent) on a currency- and portfolio-adjusted basis. The company is now targeting an EBITDA margin before special items of around 26 to 27 percent (previously: around 26 percent) on a currency-adjusted basis. Based on the aforementioned sales figure, this would now correspond to EBITDA before special items of around 12.5 billion euros (previously: around 12.0 billion euros) on a currency-adjusted basis. Core earnings per share are now expected to come in at approximately 7.30 euros (previously: approximately 7.00 euros) on a currency-adjusted basis. Free cash flow is now forecast to amount to around 2.5 billion euros (previously: around 2 billion to 2.5 billion euros) on a currency-adjusted basis. In addition, the company continues to expect year-end net financial debt of approximately 33 billion to 34 billion euros on a currency-adjusted basis. As in the overall guidance, this figure does not take into account the contractually agreed divestment of the Environmental Science Professional business.

Bayer has also prepared its guidance based on the closing exchange rates as of June 30, 2022, and the differences to the currency-adjusted targets above are as follows: the company now expects to generate sales of 50 billion to 51 billion euros (previously: approximately 47 billion euros). Based on the aforementioned sales figure, Bayer is now targeting EBITDA before special items of around 13.0 billion euros (previously: around 12.0 billion euros). Core earnings per share are now expected to come in at approximately 7.70 euros (previously: approximately 7.10 euros). In addition, the company now expects year-end net financial debt of approximately 34 billion to 35 billion euros (previously: approximately 33 billion to 34 billion euros).

Sustainability: access to innovative seeds and farming solutions

Bayer also continued to make good progress in terms of sustainability. The company is supporting the Zero Hunger Pledge, which will involve helping smallholder farmers access innovative seeds, sustainable agricultural practices and farming solutions, and thus providing them with new income-generating opportunities.

Bayer has also made important progress in a major ESG rating: MSCI ESG Research recently updated their ESG Controversies Report and lifted the red flag related to "environmental concerns over GMO crops" as well as their related allegation of a breach of the UN Global Compact Principles. This marks another important milestone in improving Bayer’s ESG rating profile.