Merrimack Pharma-Ipsen's Cancer Drug Fails In Overall Survival Measure

 

• Merrimack Pharmaceuticals Inc's 
  MACK-14.5%+ Free Alerts
   partner announced that Ipsen SA 
  IPSEY
   reported a primary analysis of the results of the Phase 3 trial of Onivyde (irinotecan liposomal injection)for second-line small cell lung cancer (SCLC). 
• The study did not meet the primary endpoint of overall survival in patients treated with Onivyde versus topotecan. 
• However, a doubling of the secondary endpoint of objective response rate (ORR) in favor of Onivyde was observed. 
• The safety and tolerability of Onivyde were consistent with its already-known safety profile, and no new safety concerns emerged. 
• The clinical study results will be communicated with the regulatory agency." 
• Ipsen indicated in its update that it would analyze the data further before making decisions about the next steps.
• "We will continue to monitor updates from Ipsen regarding the SCLC program," said Gary Crocker, Chairman, and CEO of Merrimack Pharmaceuticals. 
• Ipsen expects to publicly report its top-line data from its continuing Phase 3 study of Onivyde in first-line pancreatic ductal adenocarcinoma before the end of 2022.

https://www.benzinga.com/general/biotech/22/08/28332982/merrimack-pharma-ipsens-cancer-drug-fails-in-overall-survival-measure

Regenxbio results, corporate update

 

• RGX-314 program for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, remains on track for first BLA filing in 2024

• AFFINITY DUCHENNE™ Phase I/II trial of RGX-202 remains on track for dosing in the first half of 2023

• Announced intention to file a BLA in 2024 using the accelerated approval pathway for RGX-121 for the treatment of MPS II; pivotal program is active and enrolling patients

• $682 million in cash, cash equivalents and marketable securities of as of June 30, 2022; operational runway into 2025

• Conference call Wednesday, August 3rd at 4:30 p.m. ET

Conference Call

In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. A live audio webcast will be available at regenxbio.com/investors. Interested parties may also pre-register for the earnings conference call here. Once registration is completed, participants will be provided a dial-in number with a personalized conference code to access the call. Those who plan on participating are advised to dial in 15 minutes prior to the start time.

https://finance.yahoo.com/news/regenxbio-reports-second-quarter-2022-200500994.html

Biomarin Ups Full-year 2022 Top and Bottom-line Guidance

 "Continued strong growth of VOXZOGO and solid contributions from our other franchises drove record revenues exceeding $1 billion in the first half of the year, leading us to increase our full-year 2022 top and bottom-line guidance," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We also achieved many other significant milestones in the second quarter, including the CHMP's positive opinion for conditional marketing authorization of Roctavian, the first gene therapy to be recommended for approval in Europe for hemophilia A. Assuming a positive decision from the European Commission, BioMarin's commercial organization is ready to launch Roctavian in Europe in the third quarter. Based on the updated multi-year hemostatic efficacy observed to date following treatment with a single intravenous administration of Roctavian, we are on-track to resubmit the BLA in the U.S. to the FDA this September."

Mr. Bienaimé added, "In addition to continued robust demand for Voxzogo throughout the United States and Europe, underscored by today's significant increase in full-year 2022 guidance for Voxzogo net product revenues to between $130 million and $160 million, we were thrilled to receive commercial approval in both Japan and Australia in the quarter. As we said previously, in 2022 we expect to return to double-digit revenue growth and profitability, and we are tracking to plan as demonstrated by record revenues in both the first and second quarters of this year."

2022 Full-Year Financial Guidance (in millions, except %)

Item		Provided April 27, 2022						Revised August 3, 2022
Total Revenues		$2,050		to		$2,150		$2,060		to		$2,160
Vimizim Net Product Revenues		$650		to		$700		$655		to		$700
Naglazyme Net Product Revenues		$400		to		$440		$415		to		$450
Palynziq Net Product Revenues		$280		to		$310		$250		to		$275
Kuvan Net Product Revenues		$225		to		$250		$210		to		$235
Brineura Net Product Revenues		$145		to		$160		Unchanged
Voxzogo Net Product Revenues		$100		to		$125		$130		to		$160
Cost of Sales (% of Total Revenues)		23.0 %		to		25.0 %		22.5 %		to		24.5 %
Research and Development Expense		$665		to		$715		Unchanged
Selling, General and Administrative Expense		$790		to		$840		Unchanged
GAAP Net Income		$95		to		$135		$105		to		$145
Non-GAAP Income (1)		$350		to		$390		Unchanged

https://finance.yahoo.com/news/biomarin-announces-record-revenues-second-200300012.html

Exact Sciences offloads prostate cancer test to MDxHealth for $30M

 Exact Sciences is selling off a slice of the prostate cancer precision testing business it obtained through its $2.8 billion takeover of Genomic Health. MDxHealth is paying $25 million in cash upfront, plus $5 million in company shares.

The deal includes the Oncotype DX GPS test for predicting the aggressiveness of a prostate tumor and also most of Exact Sciences' team of urology sales and marketing professionals, the companies said in a press release.

The total cost of the deal could balloon by an additional $70 million if MDxHealth passes revenue milestones through the 2025 fiscal year. Those later payments could include a mix of cash and stock.

Oncotype DX GPS is a genomic, tissue-based test that is broadly available. The diagnostic will join MDxHealth's Select mdx and Confirm mdx tests for prostate cancer. Select mdx uses a urine sample to help identify men at risk for aggressive disease, while Confirm mdx is designed to re-examine tumor tissue for possible cancer after a patient receives a negative biopsy. MDxHealth, which focuses on urology diagnostics, also recently launched a molecular test for detecting pathogens in urinary tract infections.

The addition of Exact Sciences’ sales team will also help boost MDxHealth’s total commercial footprint and help the company market its entire testing portfolio, according to MDxHealth CEO Michael McGarrity.

Exact Sciences picked up the Oncotype DX GPS test in 2019—as well as Oncotype tests in breast and colon cancer—through its acquisition of Genomic Health, one of the largest medtech deals of that year. 

Meanwhile, Exact Sciences will continue to sell a blood test aimed at metastatic, castration-resistant prostate cancer, which it developed through a partnership with Epic Sciences. That liquid biopsy diagnostic, dubbed Oncotype DX AR-V7 Nucleus Detect, aims to predict which patients will respond to androgen receptor-targeting therapies.

This week Exact Sciences posted revenues of $521.6 million for the second quarter, a 20% gain over last year, including $154 million from its precision oncology operations and $354 million from its cancer screening business. Still, the company reported a net loss of $166.1 million, down from $176.9 million during the same period in 2021.

Exact Sciences CEO Kevin Conroy said the company is focused on getting more people tested with Oncotype and Cologuard—such as by enlisting news anchor Katie Couric for a commercial for its colorectal screening test—and that it is “prioritizing our highest impact projects to reach profitability.”

MDxHealth, meanwhile, reported revenues of $6.9 million for 2022’s second quarter, up 22% over 2021’s. The company predicts that by snagging Oncotype DX GPS—which is expected to bring in $13 million alone by the year’s end—it can push its 2022 total sales to between $40 million and $42 million, for an increase of 80% to 89%.

To help finance the deal, MDxHealth took out a $35 million loan with an affiliate of Innovatus Capital Partners, with an option for an additional $35 million in the future. At the end of June, the company reported a cash and cash equivalents balance of $40 million.

https://www.fiercebiotech.com/medtech/exact-sciences-trades-its-oncotype-prostate-cancer-test-mdxhealth-100m-deal

Moderna feeling the pressure to address monkeypox: R&D chief

 The pressure is on for Moderna to step up and save the day with a monkeypox vaccine, just as it did for COVID-19. But executives from the famed biotech insist the idea is still in the conceptual phase.

Analysts peppered Moderna’s leadership with questions on its second-quarter earnings call Wednesday about a potential monkeypox program and what Moderna needs to get that moving. The company announced plans back in May to turn its R&D engine toward the emerging viral threat but has yet to commit to anything beyond the preclinical work.

In the meantime, the World Health Organization (WHO) has sounded the alarm of the growing threat by naming monkeypox a public health emergency. Doses of the available vaccine called Jynneos from Bavarian Nordic are in short supply for the time being.

President Stephen Hoge, M.D., who serves as head of R&D at Moderna, confirmed the company is feeling the pressure of low vaccine supply and the recent WHO comments. He said the goal for Moderna would be to help with the current supply but also address the long-term public health needs for monkeypox.

“We're obviously very aware of the monkeypox concern and obviously very sensitive to recent announcements,” said Hoge. “What we are looking to do is understand if we were to develop that program, how would we move it.”

If Moderna were to find a candidate to move into further development, Hoge said the goal would be to do so “very quickly.”

“Our ability to rapidly scale has been demonstrated,” Hoge said, no doubt referring to the fast pace at which the COVID-19 shot Spikevax was developed to meet the global need of the coronavirus pandemic. “We would really be doing it to try and help generate a public health counter insurance.”

One challenge for Moderna, and anyone else looking to develop a new monkeypox vaccine, is developing the right clinical endpoints for a phase 1 clinical trial to test the candidate, Hoge noted. To do that, Moderna will need to engage with regulators to figure out exactly how to design that study, and Hoge said those conversations will be happening.

With that said, Hoge stressed that “there are other even larger public health threats right now”—including COVID, which continues to infect people across the globe.

Once Moderna has clarity on endpoints and whether it has a viable candidate for monkeypox, Hoge promised an update. But for now, “it's premature to say more.”

https://www.fiercebiotech.com/biotech/yes-moderna-feeling-pressure-address-monkeypox-rd-chief-confirms

In GSK trade secrets case, appeals court backs short sentences

 Stolen secrets are no fun, especially when they’re stolen trade secrets with big money on the line. But for two former GSK researchers, their sentences for trade secrets theft will remain somewhat brief.

After pleading guilty in 2018 to stealing trade secrets on potential cancer drugs, a unanimous panel of the U.S. Court of Appeals for the Third Circuit upheld the previously set sentences of former GSK researchers Yu Xue and Tao Li.

Xue has already served eight months, while Li has served a 59-day sentence. Prosecutors wanted 10 years for Xue and seven for Li, Reuters reports.

With the appeals ruling, the former GSK researchers will have fulfilled their sentences.

The court found that the defendants did not intend to harm GSK financially, so it decided against extending their sentences.

Until the pair were arrested in 2016, they along with others worked at GSK’s Upper Merion, Pennsylvania, facility and went on to form a pharma company called Renopharma. Xue, previously one of the top chemists at GSK who was believed to be the ringleader of the trade secrets ring, stole approximately 200 GSK documents and sent them to co-conspirators. At the time of arrest, Renopharma hadn’t made a profit or developed and sold products based on GSK’s information, the court's opinion says.

In its case against the pair, the U.S. government asserted that the intended financial loss for their former employer exceeded $1 billion.

But the appeals court found that the defendants did not use the trade secrets to complete with GSK, nor to “solicit investment, develop competing products, or develop the same products that GSK was developing." With that finding, the court upheld the sentences put in place by the District Court.

A fifth defendant was just convicted in the case in May, following the guilty pleas of the other four conspirators.

https://www.fiercepharma.com/pharma/light-sentence-holds-defendants-gsk-trade-secrets-case

Moderna recorded $800M-plus in charges for unused inventory, manufacturing capacity

 While new polls show flatlining concern about the pandemic among Americans, Moderna's COVID-19 vaccine business continues quietly churning in the background. But amid a recent decline in vaccine demand, the company has written off a sizable chunk of inventory.

In its second-quarter results announcement, Moderna said it wrote off $499 million in inventories "related to COVID-19 products that have exceeded or are expected to exceed their approved shelf-lives prior to being used." In addition, the company took a $184 million charge because of changes in prior purchase committments, and it recorded an expense of $131 million for "unutilized external manufacturing capacity."

As the pandemic receded earlier this year, media headline after headline documented falling COVID-19 vaccine demand. As one example, Moderna last month reached a deal with European officals to delay vaccine deliveries. The boosters orginially supposed to be delivered in late 2022 will now come in 2023.

For its part, Moderna on Wednesday said the second-quarter charges were "driven by a substantial reduction of our expected deliveries to COVAX and deferral of deliveries to other customers, particularly to the European Union." For accounting purposes, Moderna recorded the charges under its "cost of sales" category of expenses.

Still, Moderna is running a highly profitable business. In the second quarter, the company generated sales of $4.7 billion, a 9% increase, and pulled down net income of $2.2 billion. 

Looking forward, Moderna continues to expect strong COVID-19 vaccine sales. Last week, the company inked a $1.74 billion deal to provide 66 million doses of its new bivalent booster candidate aimed at the omicron variant.

In addition, Moderna said it's already inked 2023 vaccine supply orders with the U.K., Canada, Australia, Kuwait and Taiwan. It's discussing other potential orders with officials around the globe, execs said Wednesday.

As for 2022, Moderna is standing by its COVID-19 vaccine revenue projection of $21 billion.

Outside of the company’s only available product, Moderna is testing its mRNA vaccine tech against diseases such as flu and Zika virus. Four programs are currently in phase 3 development, including a flu mRNA vaccine and a COVID-plus-flu shot. 

https://www.fiercepharma.com/financials/moderna-pulls-47-billion-second-quarter-plus-499-write-down-expired-vaccines