On May 4, 2022 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported that it will host a webcast/conference call in conjunction with its first quarter 2022 update and financial results on Wednesday, May 11 at 4:30 p.m. ET (Press release, Acorda Therapeutics, MAY 4, 2022, View Source [SID1234613511]).

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To participate in the Webcast, please use the following pre-registration link:

View Source
If you register for the Webcast, you will have the opportunity to submit a written question for the Q&A portion of the presentation. Once you have registered, you will receive a confirmation email with Webcast/Conference Call details. For the Webcast, you will receive an email 2 hours prior to the start of the call with the link to join. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 7:30 p.m. ET on May 11, 2022 until 11:59 p.m. ET on June 10, 2022. To access the replay, please dial 1 866 813 9403 (domestic) or +44 204 525 0658 (international); reference code 258745. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On May 4, 2022 Catalyst Clinical Research, a market-leading provider of clinical research services, reported a strategic acquisition of Aptus Clinical to expand the geographic reach of the two companies, and to enhance patient access to advanced, life-changing therapies (Press release, Catalyst Clinical Research, MAY 4, 2022, View Source [SID1234613510]). Together, the two entities will become one multi-region clinical research organization with an accelerated growth trajectory in both the U.S. and Europe.

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Catalyst’s offerings will now encompass extensive and expanded clinical consulting services, as it inherits Aptus’ strong and established relationship with the Experimental Cancer Medicine Centre (ECMC) network to support trial set-up and clinical design input. Positioned at the forefront of novel cancer treatment trends, Catalyst’s active portfolio serves more than 5,300 cancer patients in need and will grow by nearly 600 patients concurrent to this acquisition.

"We are thrilled to be working with Aptus Clinical to expand our reach in the European market, serving clients around the globe to help develop innovative new therapies, improve patient outcomes and enhance our company’s long-term impact," said Catalyst Clinical Research CEO, Nick Dyer. "Our complementary service offerings and therapeutic area alignment will improve outcomes for all our clients."

"Catalyst continues to prove its capabilities as a leader in oncology clinical development and as a preferred strategic acquirer for growing businesses," said Vern Davenport, Partner at NovaQuest Capital Management and Catalyst Board Chairman. "Aptus provides Catalyst with a highly capable team of experts in Europe to accelerate its global ambitions, of which we are incredibly supportive." Catalyst Clinical Research is a portfolio company of global platform NovaQuest Capital Management, LLC.

Formed in 2014 by three former AstraZeneca clinical development experts, Aptus Clinical has rapidly established itself as a specialist CRO offering flexible clinical research solutions in oncology, cell and gene therapies and rare diseases. Together the companies, operating as Catalyst Oncology, will continue to deliver on a portfolio of more than 100 active oncology studies via a network of 500 keenly involved investigator sites. Additional studies outside the Oncology therapeutic area will be delivered under the Catalyst Flex solution and operating models.

"We are proud to partner with Catalyst to strengthen our shared commitment to delivering cutting edge science to patient populations who are in desperate need of new therapies", said Aptus Clinical CEO, Dr. Steve McConchie. "We firmly believe Aptus Clinical and Catalyst are better together and look forward to combining our decades of experience in developing and delivering high quality clinical trials to support our combined organisation to become a significant global provider of innovative clinical research solutions."

Combined headcount for the unified company will now exceed 800 staff members. Catalyst will also work to leverage Aptus’ strategic site relationships integrating them into its own site network spanning North America and Europe. The clinical research organization joined forces with Triangle Biostatistics in 2019, and then with Ce3 in 2020. Although this is Catalyst’s third M&A transaction, it is its first international union that will accelerate and expand the companies’ geographic reach greatly.

On May 4, 2022 Fresenius Medical Care reported When we see what is happening in Ukraine, it is again above all the human tragedy that leaves us deeply saddened (Press release, Fresenius, MAY 4, 2022, View Source [SID1234613509]). I am incredibly thankful and proud of all who continue to work tirelessly to ensure patient care and the holding up of our local operations under these outstandingly difficult circumstances. Although it is difficult to talk about numbers with these images in mind, I have to say that in addition, Omicron has affected the quarter heavily. This resulted in high excess mortality among our patients and significantly elevated labor costs in the U.S. to manage isolation clinics and shifts. We were able to compensate this and delivered the quarter in line with our expectations. Based on a strong decline in excess mortality in February and March, we confirm our financial targets for 2022."
Higher than expected COVID-19-related excess mortality at the beginning of the year

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COVID-19-related excess mortality among Fresenius Medical Care’s patients amounted to approximately 2,310 in the first quarter of 2022 (Q1 2021: ~3,200; Q2 2021: ~1,900;
Q3 2021: ~2,900; Q4 2021: ~2,0003). It significantly declined in February and March in line with infection rates, but on a quarterly basis still exceeded the originally anticipated level. This resulted in an increased need for isolation clinics and shifts and limited the Company’s ability to mitigate the impacts from labor shortage and wage inflation in the U.S. market.
COVID-19-related excess mortality accumulated to approximately 9,000 patients over the past twelve months and to approximately 22,600 since the start of the pandemic.
The overall estimated adverse effect of accumulated COVID-19-related excess mortality on organic growth in the Health Care Services business amounted to around 290 basis points in the first quarter.

1 2021: costs related to the FME25 program; 2022: costs related to the FME25 program and impacts related to the war in Ukraine
2 Net income attributable to shareholders of Fresenius Medical Care AG & Co. KGaA
3 Historical excess mortality updated for late entries

War in Ukraine impacting business development

The war in Ukraine is affecting Fresenius Medical Cares’ dialysis operations and patient care in the country itself, but also caused higher bad debt expenses in Russia and Ukraine. The direct adverse effect of the war in Ukraine amounted to EUR 22 million at operating income level in the first quarter and is treated as a special item. Fresenius Medical Care will continue to monitor closely the potential effects of the war as well as the general impact of the challenging inflationary macroeconomic environment.

First quarter earnings development in line with expectations

Revenue increased by 8% to EUR 4,548 million (+3% at constant currency, +2% organic).

Health Care Services revenue increased by 8% to EUR 3,607 million (+3% at constant currency, +1% organic). At constant currency, this was mainly driven by organic growth, which was achieved despite the adverse impact of COVID-19, the partial reversal of an accrual related to a revenue recognition adjustment for accounts receivable in legal dispute and contributions from acquisitions.

Health Care Products revenue increased by 6% to EUR 941 million (+3% at constant currency, +3% organic). Constant currency growth was mainly driven by higher sales of in-center disposables and renal pharmaceuticals. This was partially offset by lower sales of machines for chronic treatment.

Operating income decreased by 27% to EUR 348 million (-30% at constant currency), resulting in a margin of 7.6% (Q1 2021: 11.3%). Operating income excluding special items, i.e. costs incurred for FME25 and the impacts related to the war in Ukraine, declined by 15% to EUR 403 million (-19% at constant currency), resulting in a margin of 8.9% (Q1 2021: 11.3%). At constant currency, the decline was mainly due to higher labor costs, adverse COVID-19-related effects, as well as inflationary and supply chain cost increases. These effects were only partially mitigated by the partial reversal of an accrual related to a revenue recognition adjustment for accounts receivable in legal dispute.

Net income2 decreased by 37% to EUR 157 million (-39% at constant currency). Excluding special items, net income declined by 20% to EUR 200 million (-23% at constant currency), mainly due to the mentioned negative effects on operating income.

Basic earnings per share (EPS) decreased by 37% to EUR 0.54 (-39% at constant currency). EPS excluding special items declined by 20% to EUR 0.68
(-23% at constant currency).

Cash flow development

In the first quarter, Fresenius Medical Care generated EUR 159 million of operating cash flow (Q1 2021: EUR 208 million), resulting in a margin of 3.5% (Q1 2021: 4.9%). The decrease was mainly due to continued recoupment of the U.S. government’s payments received in 2020 under the CARES Act and a decrease in net income, partially offset by a favorable impact from trade accounts and other receivables.

Free cash flow4 amounted to EUR -1 million (Q1 2021: EUR 29 million) in the first quarter, resulting in a margin of 0.0% (Q1 2021: 0.7%).

Regional developments

In North America, revenue increased by 9% to EUR 3,171 million (+2% at constant currency, +0% organic). At constant currency, this was mainly driven by organic growth in the Health Care Product business and the reversal of an accrual related to a revenue recognition adjustment for accounts receivable in legal dispute. This was partially offset by the adverse COVID-19 impact on the Health Care Services business.

Operating income in North America decreased by 24% to EUR 304 million (-29% at constant currency), resulting in a margin of 9.6% (Q1 2021: 13.7%). At constant currency, the decline in operating income was mainly due to higher labor costs, the adverse impact of COVID-19, inflationary and supply chain cost increases as well as costs related to FME25. This was only partially offset by the partial reversal of an accrual related to a revenue recognition adjustment for accounts receivable in legal dispute.

Revenue in the EMEA region increased by 1% to EUR 674 million in the first quarter (+3% at constant currency, +2% organic). At constant currency, this was mainly due to organic growth in the Health Care Services business, which was achieved despite the negative impact of COVID-19.

Operating income in EMEA decreased by 23% to EUR 61 million (-19% at constant currency), resulting in a margin of 9.1% (Q1 2021: 11.9%). The decline was mainly due to the impact related to the war in Ukraine.

4 Net cash provided by / used in operating activities, after capital expenditures, before acquisitions, investments, and dividends

In Asia-Pacific, revenue increased by 8% to EUR 507 million (+4% at constant currency, +4% organic). At constant currency, this was mainly driven by organic growth in the Health Care Products business.

Operating income increased by 16% to EUR 99 million (+14% at constant currency), resulting in a margin of 19.5% (Q1 2021: 18.1%). At constant currency, this was mainly due to a gain from the sale of clinics, favorable currency transaction effects and growth in the Health Care Products business.

Latin America revenue increased by 15% to EUR 183 million (+15% at constant currency, +16% organic), mainly driven by strong organic growth in both the Health Care Services and Health Care Products business.

Operating income improved by 68% to EUR 11 million (+51% at constant currency), resulting in a margin of 6.1% (Q1 2021: 4.2%). This was mainly due to a favorable currency transaction effect, which was partially offset by inflationary cost increases.

Patients, clinics and employees

As of March 31, 2022, Fresenius Medical Care treated 343,493 patients in 4,153 dialysis clinics worldwide and had 122,635 employees (full-time equivalents) globally, compared to 124,995 employees as of March 31, 2021.

Outlook

Based on the results for the first quarter, which were in line with the Company’s expectations, Fresenius Medical Care confirms its financial targets for 2022. The earnings improvement will be driven by expected business growth, PPE cost reduction and FME25 savings. The Company expects revenue and net income to grow at low to mid-single digit percentage rates in FY 2022.5

5 These targets are based on the 2021 results excluding the costs related to FME25 of EUR 49 million (for Net Income). They are based on the assumptions outlined in the Press Release on the Q4 and FY 2021 results (Feb. 22, 2022), in constant currency and exclude special items. Special items include further costs related to FME25, the impacts related to the war in Ukraine, and other effects that are unusual in nature and have not been foreseeable or not foreseeable in size or impact at the time of giving guidance.

Conference call

Fresenius Medical Care will host a conference call to discuss the results of the first quarter 2022 on May 4, 2022 at 3:30 p.m. CEST / 9:30 a.m. EDT. Details will be available on the Fresenius Medical Care website in the "Investors" section. A replay will be available shortly after the call.

On May 4, 2022 Fresenius Kabi reported that closed the acquisition in March of Ivenix, Inc. ("Ivenix"), a specialized infusion therapy company (Press release, Fresenius, MAY 4, 2022, View Source [SID1234613508]). Ivenix adds a next-generation infusion therapy platform for the significant U.S. market to Fresenius Kabi’s portfolio and provides the company with key capabilities in hospital connectivity. The combination of Ivenix’s leading hardware and software products with Fresenius Kabi’s offering in intravenous fluids and infusion devices will create a comprehensive and leading portfolio of premium products, forming a strong basis to enable sustainable growth in the high-value MedTech space. The purchase price is a combination of US$240 million upfront payment and milestone payments, strictly linked to the achievement of commercial and operating targets.

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On May 4, 2022 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported financial results for the first quarter of 2022 and recent business achievements (Press release, Ionis Pharmaceuticals, MAY 4, 2022, View Source [SID1234613507]).

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"We are off to a strong start this year highlighted by progress in our rich late- and mid-stage pipeline. We remain on track for data from the NEURO-TTRansform study of eplontersen in patients with hereditary ATTR polyneuropathy by mid-year. Assuming positive data, we plan to file for regulatory approval by the end of this year. We recently increased the size and duration of our CARDIO-TTRansform study of eplontersen in patients with ATTR cardiomyopathy. Our aim is to generate even more robust data and ensure a highly positive study outcome to successfully compete in this growing and dynamic market," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "We also reported positive data from two potential best-in-class medicines. In the Phase 2b ETESIAN study, ION449, our investigational medicine targeting PCSK9, demonstrated robust LDL-C and PCSK9 reductions in statin-treated patients with hypercholesterolemia. ION449 was generally well tolerated in this study. Additional positive data from the Phase 2 study of donidalorsen demonstrated significant improvements in quality of life in people with hereditary angioedema. Looking ahead, we expect to provide additional key mid-stage data readouts and updates on our technology advancements. These upcoming catalysts, together with our recent achievements, position us well to continue to drive substantial growth and value for all stakeholders."

First Quarter 2022 Summary Financial Results

On track to achieve 2022 financial guidance, based on the following first quarter results:

$142 million in total revenues
$173 million of operating expenses on a non-GAAP basis(1) and $199 million on a GAAP basis
$39 million net loss on a non-GAAP basis(1) and $65 million on a GAAP basis
$2.1 billion of cash and short-term investments
"A key element of Ionis’ financial strength is our ability to generate substantial revenue from multiple diverse sources on a sustained basis. Our first quarter financial results in which revenues grew more than 25 percent year over year were an excellent example of this. We generated revenues from our marketed products, including SPINRAZA, and from numerous partnered medicines as they advanced. Our first quarter financial results also reflect our investments in our rich late-stage pipeline and in activities to prepare for our launches of eplontersen, olezarsen and donidalorsen," said Elizabeth L. Hougen, chief financial officer of Ionis. "We are on track to meet our 2022 financial guidance. With $2.1 billion of cash, we are well capitalized with the resources we need to continue investing in our large agenda to drive substantial future growth."

Recent Marketed Products Highlights

SPINRAZA: the global market leader for the treatment of spinal muscular atrophy (SMA) patients of all ages

$473 million in worldwide SPINRAZA sales in the first quarter
Biogen provided updates from the ASCEND, RESPOND and NURTURE studies of SPINRAZA at the Muscular Dystrophy Association (MDA) Clinical and Scientific conference and the American Academy of Neurology (AAN) annual meeting
TEGSEDI and WAYLIVRA: important medicines approved for the treatment of patients with polyneuropathy caused by hereditary TTR amyloidosis and familial chylomicronemia syndrome, respectively

Continued to progress into new and existing markets in Europe and Latin America in the first quarter through Swedish Orphan Biovitrum AB (Sobi) and PTC Therapeutics, respectively
First Quarter 2022 and Recent Events

Advancing Ionis’ near-term commercial opportunities toward the market

Increased study size and duration in the Phase 3 CARDIO-TTRansform study of eplontersen in patients with ATTR cardiomyopathy with the aim to generate even more robust data and ensure a highly positive study outcome to successfully compete in this growing and dynamic market. Data from this study are expected in the first half of 2025
The U.S. FDA granted orphan drug designation to eplontersen for the treatment of patients with ATTR
Published positive data from the Phase 2 study of olezarsen in patients with hypertriglyceridemia and either at high risk for or with established cardiovascular disease in the European Heart Journal
Initiated a study of olezarsen in patients with hypertriglyceridemia to support the broad Phase 3 program
Published positive data from the Phase 2 study of donidalorsen in patients with hereditary angioedema (HAE) in the New England Journal of Medicine
Presented additional positive data from the Phase 2 study of donidalorsen in patients with HAE at the American Academy of Allergy, Asthma and Immunology (AAAAI) annual meeting
Advancing Ionis’ leading cardiovascular disease franchise

AstraZeneca presented positive data from the Phase 2b ETESIAN study of ION449 (AZD8233) targeting PCSK9 in statin treated patients with dyslipidemia at the American College of Cardiology (ACC) annual scientific session
Achieved full enrollment in the Phase 2b study of IONIS-AGT-LRx in patients with treatment-resistant hypertension, with data expected in the second half of 2022
Advancing Ionis’ leading neurological disease franchise

Roche plans to initiate a new Phase 2 study of tominersen in patients with Huntington’s disease based on findings from a post-hoc analysis of the GENERATION-HD1 study
Biogen initiated a Phase 1/2 study of ION260 (BIIB132) targeting ataxin-3 (ATXN3) in patients with spinocerebellar ataxia type 3 (SCA3), resulting in an $8 million milestone payment from Biogen
Biogen advanced the Phase 1/2 study of ION859 (BIIB094) targeting LRRK2 in patients with Parkinson’s disease, resulting in a $10 million milestone payment from Biogen
Announced the discontinuation of IONIS-C9Rx (BIIB078) due to lack of patient benefit demonstrated in the Phase 1/2 study in patients with C9orf72-ALS
2022 Pipeline Milestones(2)

Anticipated 2022 Regulatory Updates

Program

Anticipated Indication

Regulatory Action

H1

H2

Eplontersen

ATTRv polyneuropathy

NDA filing

Anticipated Key 2022 Data Readouts

Program

Data Readout

Anticipated Indication

H1

H2

Tominersen

Phase 3 post hoc

Huntington’s disease

✓

ION449 (PCSK9)

Phase 2b (ETESIAN)

Cardiovascular disease

Donidalorsen

Phase 2

HAE

IONIS-C9Rx (BIIB078)

Phase 1/2

C9-ALS

Tofersen

Phase 3 OLE

SOD1-ALS

Eplontersen

Phase 3

ATTRv polyneuropathy

IONIS-AGT-LRx

Phase 2b

Treatment-resistant hypertension

Fesomersen (FXI)

Phase 2b

Thrombosis

Bepirovirsen (HBV)

Phase 2b

Hepatitis B virus infection

Donidalorsen

Phase 2 OLE

HAE

Cimdelirsen (GHR)

Phase 2 (monotherapy)

Acromegaly

Anticipated Key 2022 Study Initiations

Program

Phase

Anticipated Indication

H1

H2

Sapablursen

2

Polycythemia vera

IONIS-MAPTRx (BIIB080)

2

Alzheimer’s disease

ION904 (AGT)

2

Uncontrolled hypertension

ION717 (PRNP)

1/2

Prion disease

Anticipated Key 2022 Technology Advancements

Program

Anticipated Advancement

H1

H2

SMA

Advance follow-on program

Muscle LICA

Advance into preclinical development (IND-supporting)

MsPA Backbone

Advance into preclinical development (IND-supporting)

✓ = achieved • = planned

All non-GAAP amounts referred to in this press release exclude non-cash compensation
expense related to equity awards. In 2021 all non-GAAP amounts also excluded expenses
related to the Akcea Merger and restructured commercial operations and the related tax
effects. Please refer to the detailed reconciliation of non-GAAP and GAAP measures, which is
provided later in this press release.

The Company’s revenue in the first quarter of 2022 increased more than 25 percent compared to the same period last year. The increase was driven by significant partner payments across multiple partnered programs, including $20 million from AstraZeneca for its share of the global Phase 3 program costs for eplontersen. Since Ionis is conducting the ongoing Phase 3 program for eplontersen, Ionis recognizes revenue for the reimbursements it receives from AstraZeneca for development expenses in the same period Ionis recognizes the related development expenses. Refer to the detailed table of costs and reimbursements for the eplontersen collaboration provided later in this release. The Company also earned $40 million from Biogen for advancing several neurology disease programs.

The Company successfully completed the transition of its TEGSEDI and WAYLIVRA operations in the EU and North America to Sobi in the first and second quarters of 2021, respectively. The decrease in TEGSEDI and WAYLIVRA revenue in the first quarter of 2022 compared to the same period last year was due to the shift from product sales to distribution fees based on net sales generated by Sobi. As part of the transition, Ionis restructured its commercial operations in 2021 resulting in substantial cost savings.

Operating Expenses

Ionis is advancing a large late-stage pipeline and as a result, its non-GAAP operating expenses increased in the first quarter of 2022 compared to the same period in 2021. Higher R&D expenses were driven by the expanded number of Phase 3 studies the Company is conducting, which doubled over the course of 2021 from three to six studies. Lower SG&A expenses were largely due to the substantial savings Ionis achieved from integrating Akcea and restructuring its commercial operations. These savings were offset in part by the investments Ionis made in advancing its go-to-market activities for its near-term commercial opportunities.

Net Loss

Ionis’ net loss in the first quarter of 2022 decreased compared to the same period in 2021 primarily related to the changes in revenue and operating expenses, as discussed above.

Balance Sheet

As of March 31, 2022 and December 31, 2021, Ionis had cash, cash equivalents and short-term investments of $2.1 billion. Ionis’ debt obligations and working capital did not change significantly from December 31, 2021 to March 31, 2022.

Webcast

Ionis will conduct a webcast today at 11:30 a.m. Eastern time to discuss this announcement and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.