Vertex reiterates guidance after Q1 results

 Product revenues of $1.72 billion, a 14% increase compared to Q1 2020-

- Company advancing clinical programs in six additional diseases beyond cystic fibrosis-

-Multiple Phase 2 proof-of-concept study results expected in 2021 -

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the first quarter ended March 31, 2021 and reiterated full-year 2021 guidance for product revenues.

"In CF, our goal is that all eligible patients have access to and can benefit from CFTR modulators. In the first quarter, we continued to make significant progress towards this goal, and in so doing again delivered strong revenue and earnings growth," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "Beyond CF, we have also seen continued significant progress across our broad pipeline, including advancement of VX-548 to Phase 2 in acute pain, initiation of the Phase 1/2 clinical trial with VX-880 in type 1 diabetes and completion of enrollment and dosing in our Phase 2 proof-of-concept study with the AAT corrector, VX-864. The recent amendment of our agreement with CRISPR Therapeutics for the CTX001 program further enhances our leadership position in cell and genetic therapies and we look forward to completing enrollment of our ongoing trials for CTX001 in sickle cell disease and beta thalassemia this year and bringing this first-in-class treatment to patients with these devastating diseases as soon as possible."

First-Quarter 2021 Financial Highlights

		Three Months Ended March 31,										%
		2021					2020					Change
		(in millions, except per share amounts)
Product revenues, net		$	1,723				$	1,515				14	%
TRIKAFTA/KAFTRIO		$	1,193				$	895
SYMDEKO/SYMKEVI		$	125				$	173
ORKAMBI		$	219				$	234
KALYDECO		$	186				$	213
GAAP Operating income		$	888				$	720				23	%
Non-GAAP Operating income		$	1,003				$	877				14	%
GAAP Net income		$	653				$	603				8	%
Non-GAAP Net income		$	781				$	674				16	%
GAAP Net income per share - diluted		$	2.49				$	2.29				9	%
Non-GAAP Net income per share - diluted		$	2.98				$	2.56				16	%

Product revenues increased 14% compared to the first quarter of 2020, primarily driven by the uptake of KAFTRIO in Europe and continued performance of TRIKAFTA in the U.S. Net product revenues in the first quarter of 2021 increased 6% to $1.25 billion in the U.S. and increased 43% to $470 million outside the U.S., compared to the prior year.

Conference Call and Webcast

The company will host a conference call and webcast today at 5:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section under "Events and Presentations." To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website.

https://finance.yahoo.com/news/vertex-reports-first-quarter-2021-200100624.html

Veklury drives Gilead Q1 sales

 First Quarter 2021 Product Sales Increased 16% Year-Over-Year Primarily Driven by Veklury

Returned $1.2 Billion of Cash to Shareholders in First Quarter 2021 Through Dividends and Share Repurchases

Gilead Sciences, Inc. (Nasdaq: GILD) announced today its results of operations for the first quarter 2021.

"We have made strong progress in this first quarter, with our new partnership with Merck in long-acting HIV therapies, two newly approved indications in the U.S. for Trodelvy in metastatic triple-negative breast cancer and metastatic urothelial cancer, and the addition of Hepcludex to our portfolio," said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. "2021 is a pivotal year for Gilead, with key milestones across our virology and oncology portfolios. We’re looking forward to advancing our pipeline of promising therapies in the coming months."

First Quarter 2021 Financial Results

• Total first quarter 2021 revenue of $6.4 billion increased 16% compared to the same period in 2020, primarily due to Veklury® (remdesivir) sales, Cell Therapy growth with Yescarta® (axicabtagene ciloleucel) and the U.S. launch of Tecartus® (brexucabtagene autoleucel) in the third quarter 2020, the first full quarter recognition of Trodelvy® (sacituzumab govitecan-hziy 180 mg) sales, and Hepatitis B virus ("HBV") growth with Vemlidy® (tenofovir alafenamide 25 mg).

• Diluted Earnings Per Share ("EPS") increased 12% to $1.37 for the first quarter 2021 compared to the same period in 2020, primarily driven by revenue growth, partially offset by fair value loss adjustments related to Gilead’s equity investment in Galapagos NV ("Galapagos") and lower interest income.

• Non-GAAP diluted EPS increased 24% to $2.08 for the first quarter 2021 compared to the same period in 2020, primarily due to higher operating income and lower effective tax rate, offset by lower interest income.

• As of March 31, 2021, Gilead had $6.2 billion of cash, cash equivalents and marketable debt securities compared to $7.9 billion as of December 31, 2020.

• During the first quarter 2021, Gilead generated $2.6 billion in operating cash flow.

• During the first quarter 2021, Gilead repaid $1.3 billion of debt, utilized $1.3 billion on acquisitions, net of cash acquired (including in-process research and development ("IPR&D")), paid cash dividends of $917 million and utilized $309 million on repurchases of common stock.

https://finance.yahoo.com/news/gilead-sciences-announces-first-quarter-200100038.html

Lung Cancer Survival Continues to Rise With Immunotherapy

 Twice as many patients with advanced lung cancer carrying high PD-L1 expression lived 5 years or longer if they started treatment with pembrolizumab (Keytruda) instead of chemotherapy, long-term follow-up from a randomized trial showed.

Five-year overall survival (OS) was 31.9% in pembrolizumab-treated patients and 16.3% for patients who received chemotherapy. Median OS also was twice as long with pembrolizumab, 26.3 months versus 13.4 months (HR 0.62, 95% CI 0.48-0.81).

The advantage for treatment with the PD-1 inhibitor emerged despite the fact that two-thirds of patients in the chemotherapy group eventually crossed over to the immunotherapy arm, which might have led to underestimation of the magnitude of benefit with pembrolizumab, investigators reported in the Journal of Clinical Oncology.

"This high effective crossover rate may have reduced the observed treatment effect for pembrolizumab versus chemotherapy given that immunotherapy agents (including pembrolizumab) have demonstrated improved OS over chemotherapy in the second line or later setting," said Martin Reck, MD, PhD, of Lung Center Grosshansdorf in Germany, and colleagues. "In a prior analysis we showed that when OS for patients in the chemotherapy group of KEYNOTE-024 was adjusted for crossover ... the HR for OS was 0.49."

"Pembrolizumab provided a durable and clinically relevant long-term OS benefit versus chemotherapy as first-line therapy for metastatic NSCLC [non-small cell lung cancer] with PD-L1 TPS [tumor proportion score] of at least 50% and is a standard-of-care therapy in this setting," they concluded.

The results confirmed and strengthened observations from a previously reported analysis of 3-year survival. The results also are consistent with the KEYNOTE-001 trial, a nonrandomized trial that showed a 5-year OS of 29.6% with single-agent pembrolizumab in treated and untreated metastatic NSCLC.

The KEYNOTE-024 trial compared pembrolizumab and platinum-based chemotherapy as the first treatment for patients with metastatic NSCLC and PD-L1 expression ≥50%. Investigators in the global trial randomized 305 patients to single-agent pembrolizumab or a platinum-based chemotherapy combination. The primary endpoint was progression-free survival (PFS), and OS was a key secondary endpoint.

As previously reported, the pembrolizumab arm had a median PFS of 10.3 months versus 6.0 months for the chemotherapy arm (P<0.001). Other secondary endpoints also favored the pembrolizumab arm, including investigator-assessed objective response rate (46.1% vs 31.1%), median duration of response (29.1 vs 6.3 months), median PFS2 (24.1 vs 8.5 months), and median PFS2 at 3 years (39.5% vs 15.0%).

The authors said no new safety signals emerged with longer follow-up in the trial. Treatment-related adverse events (TRAEs), serious TRAEs, and fatal TRAEs (two with pembrolizumab and three with chemotherapy) occurred in a similar proportion of patients in each group.

Immunotherapy's impact on lung cancer survival has been "amazing," said Roy Herbst, MD, PhD, of Yale Cancer Center in New Haven, Connecticut.

"I started doing this work 25 years ago when we were using platinum and taxanes and ifosfamide, and the 1-year survival was 20% or 30% and 2-year survival almost zero, and now we have 5-year overall survival of almost 32%," he told MedPage Today. "This really tells us that we are making amazing progress, and there's a group of patients that I would say are cured from lung cancer."

"These drugs work amazingly well in a reasonable subset of patients and they work okay for the rest of the patients," Herbst added. "We have to figure out how to make it work better for all lung cancer patients and for other types of solid tumors as well."

A key limitation of immunotherapy is the lack of reliable biomarkers to guide treatment to the patients who are most likely to benefit. Future research should include a focus on "personalizing" immunotherapy, as has been done with targeted therapy.

"The problem is we don't know a priori who those patients [who do well] are going to be," said Herbst. "Now is totally the time to personalize immunotherapy. We've taken all of these years personalizing targeted therapy, and now we have seven or eight targets and we're using them quite specifically. We need to do the same thing with immunotherapy so we can help the other 70% or so of patients who don't survive for 5 years."

Disclosures

The KEYNOTE-024 trial was supported by Merck.

Reck disclosed relevant relationships with Lilly, Merck, Bristol Myers Squibb, AstraZeneca, Boehringer Ingelheim, Pfizer, Novartis, Roche/Genentech, AbbVie, Amgen, Mirati Therapeutics, Samsung Bioepis, Celgene, and Pfizer.

Primary Source

Journal of Clinical Oncology

Source Reference: Reck M, et al "Five-year outcomes with pembrolizumab versus chemotherapy for metastatic non-small cell lung cancer with PD-L1 tumor proportion score ≥50%" J Clin Oncol 2021; DOI: 10.1200/JCO.21.00174.

https://www.medpagetoday.com/hematologyoncology/lungcancer/92329

Woodcock’s next target: trials with no summary results

 If the US FDA’s acting commissioner, Janet Woodcock, wants to be seen to be getting tough with biopharma then she appears to have pinpointed her next target: companies that do not post on clinicaltrials.gov summary results of completed studies. Yesterday the agency called out one such company, Acceleron, which it has sent a notice of noncompliance for this misdemeanour, having earlier issued it a pre-notice of noncompliance. However, the numbers show the FDA to be nowhere close to scratching the surface of this problem. Out of over 67,000 industry-funded studies completed over a year ago, the maximum time usually allowed before results must be submitted, 63% have no results posted on clinicaltrials.gov. Yet amazingly the FDA has sent only 40 pre-notices of noncompliance. And the Acceleron trial in question, the Dart study of dalantercept, is hardly high-level, the project having been discontinued in 2017. The FDA’s other show of toughness, a three-day adcom scrutinising “dangling” approvals of oncology drugs with failed confirmatory trials, has so far had the effect of strongly endorsing the first four indications. The adcom concludes after today's discussion of Keytruda in gastric and Keytruda and Opdivo in liver cancer.

Completed studies with no summary results posted on clinicaltrials.gov
	Completed	With results posted	Pct without results
All trials	201,816	40,921	80%
All with Apr 2020 primary completion	178,641	40,642	77%
Funded by industry	74,968	24,785	67%
Industry-funded, with Apr 2020 primary completion	67,175	24,656	63%
Source: clinicaltrials.gov. https://www.evaluate.com/vantage/articles/news/snippets/woodcocks-next-target-trials-no-summary-results

Ocular TherapeutixTo Present at 2021 Association for Research in Vision and Ophthalmology Meet

Ocular Therapeutix, Inc. (NASDAQ:OCUL), a biopharmaceutical company focused on the formulation, development, and commercialization of innovative therapies for diseases and conditions of the eye, announced multiple scientific presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. With the ARVO 2021 Annual Meeting being moved to a virtual format, video-recorded presentations are scheduled to become available online at ARVOLearn on May 1, 2021.

"We are pleased to be presenting data on our pre-clinical and clinical programs at this year’s annual meeting, including updated data on two of our key programs, OTX-TKI for the treatment of wet AMD and OTX-TIC for the reduction of intraocular pressure in patients with primary open-angle glaucoma or ocular hypertension," commented Michael Goldstein, MD, MBA, President, Ophthalmology and Chief Medical Officer of Ocular Therapeutix.

Online presentations are expected to be hosted in the Media library catalog within ARVOLearn.

https://finance.yahoo.com/news/ocular-therapeutix-present-pre-clinical-120000910.html

AtriCure’s EPi-Sense Afib Treatment Approved by FDA

 FDA approval results in the only label of its kind for more than 3 million patients in the United States, significantly expanding AtriCure’s addressable market

Superiority trial showed a 29% difference in effectiveness at 12 months and a 35% difference in effectiveness at 18 months for long-standing persistent Afib patients.

Study also showed improved Electrophysiology Lab efficiency

https://finance.yahoo.com/news/atricure-epi-sense-system-approved-110000221.html

CVS Health Launches $100 Million Venture Fund

CVS Health Ventures to focus on high-potential, early-stage companies that strive to make health care more accessible and affordable for consumers

CVS Health (NYSE: CVS) today announced the launch of CVS Health Ventures, a dedicated corporate venture capital fund that will invest in and partner with high-potential, early-stage companies focused on making health care more accessible, affordable, and simpler.

The fund will initially launch with $100 million allocated for investments and will focus on companies with the potential for technology-enabled innovation and disruption in digital health care that are anchored in CVS Health's core strategy. CVS Health Ventures will build relationships with early-stage companies via investment as well as by offering expertise and insights from CVS Health's unique perspective.

CVS Health has already made more than 20 direct investments through the CVS and Aetna businesses. These investments have delivered consistently strong returns and partnerships. Current investments include Unite Us, a technology platform that connects health care and social services providers, and LumiraDx, an innovative point-of-care diagnostic platform.

Additional information on CVS Health Ventures and its leadership team can be found at cvshealth.com/ventures.

https://finance.yahoo.com/news/cvs-health-launches-100-million-130000279.html