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Friday, January 5, 2024

Avidity: Corporate Priorities and Catalysts for Next Stage of Growth

  Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced its 2024 corporate priorities and catalysts for the next stage of growth. In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1). The robust data package of AOC 1001 from the Phase 1/2 MARINA® trial and open-label extension study, MARINA-OLE™, has demonstrated consistent improvements across multiple functional endpoints including myotonia, muscle strength and mobility, and long-term favorable safety and tolerability results in people living with DM1. Avidity also plans to report data in 2024 from all three of its ongoing clinical programs targeting three distinct rare muscle diseases: DM1, Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44) and facioscapulohumeral muscular dystrophy (FSHD) while advancing its cardiology programs toward clinical development. In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.

2024 Upcoming Milestones & Key Highlights

  • Initiation of global Phase 3 HARBOR™ trial of AOC 1001 for DM1 in mid-2024
  • Data readouts from all three clinical programs for three distinct muscle diseases
    • Q1 2024: First look at AOC 1001 efficacy data from MARINA-OLE™ trial in people living with DM1
    • Q2 2024: AOC 1020 data from a preliminary assessment in approximately half of patients in Phase 1/2 FORTITUDE™ trial in FSHD
    • 2H 2024: First look at AOC 1044 data in people living with DMD44 from Phase 1/2 EXPLORE44™ trial
  • Dose-escalation of remaining study participants from 2 mg/kg to 4 mg/kg of AOC 1001 in the MARINA-OLE trial
  • Advance wholly-owned and partnered cardiology programs toward clinical development following recent expansion of cardiology collaboration with Bristol Myers Squibb
  • Strong cash balance with funding through 2025

Bluebird Secures Second Big Outcomes-Based Coverage Contract for Lyfgenia

 A month after winning the FDA’s approval for its sickle cell disease gene therapy Lyfgenia (lovotibeglogene autotemcel), bluebird bio has secured its second outcomes-based coverage deal, the biotech disclosed Thursday in an SEC filing.

The agreement pushes the cumulative total of covered U.S. patients for Lyfgenia to approximately 200 million people, the company noted in its filing.

Bluebird previously signed a similar outcomes-based agreement in December 2023, a week after Lyfgenia’s FDA approval. The company did not name the payer but said that it represents approximately 100 million covered patients in the U.S.

At the time, bluebird also revealed in an SEC filing that it was in advanced talks with other large commercial payers and with more than 15 Medicaid agencies, representing some 80% of sickle cell disease (SCD) patients in the U.S.

Bluebird said it will provide more details regarding the commercial launch of Lyfgenia at next week’s 42nd annual J.P. Morgan Healthcare Conference.

Lyfgenia is a gene therapy that delivers a functional copy of the β-globin gene directly into the blood stem cells, allowing patients to produce their own anti-sickling hemoglobin and reducing the overall proportion of sickled hemoglobin.

The FDA approved Lyfgenia on Dec. 8, 2023, authorizing its use to treat SCD patients 12 years and older who have a history of vaso-occlusive events. Despite the regulatory win, however, bluebird’s stock tanked nearly 35% that same day.

Driving the market’s skepticism of Lyfgenia is its hefty wholesale acquisition price of $3.1 million. By comparison, Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel)—a competing SCD gene therapy approved by the FDA on the same day as Lyfgenia—will cost $2.2 million. Lyfgenia also carries a boxed warning, flagging cases of hematologic malignancies in treated patients, while Casgevy does not.

Seeking to bolster its cash position, bluebird announced weeks after Lyfgenia’s approval that it was raising $250 million through two funding opportunities: a $150 million underwritten public offering and a $100 million accounts receivable factoring agreement.

As of the end of September 2023, bluebird had around $227 million in cash, cash equivalents, marketable securities and restricted cash, which is enough to support the company into the second quarter of this year, according to its third-quarter 2023 financial report.

https://www.biospace.com/article/bluebird-secures-second-big-outcomes-based-coverage-contract-for-lyfgenia/

Forget Rates, Now Worry About the Fed Unwinding Its Balance Sheet

 

  • Discussions come as funding market showing signs of angst
  • Minutes show officials trying to determine unwind guidance

The memory of a corner of the funding markets blowing up more than four years ago is still seared into the brains of many market participants. That episode will also be back of mind for the Federal Reserve as it attempts to halt its balance sheet runoff again.

This past week, the minutes from the latest Federal Open Market Committee meeting revealed that the Fed is already thinking about its balance sheet. But the last time the Fed attempted to slowly halt the process of unwinding its balance sheet — a process known as quantitative tightening, or QT, its efforts lasted only months before ructions in 2019 in the funding markets prompted a re-think.

https://www.bloomberg.com/news/articles/2024-01-05/forget-rates-now-worry-about-the-fed-unwinding-its-balance-sheet

Iran’s oil trade with China stalls as Tehran demands higher prices

 China’s oil trade with Iran has stalled as Tehran withholds shipments and demands higher prices from its top client, tightening cheap supply for the world’s biggest crude importer, refinery and trade sources said.

The cutback in Iranian oil, which makes up some 10 per cent of China’s crude imports and hit a record in October, could support global prices and squeeze profits at Chinese refiners.

The abrupt move, which one industry executive called a “default”, could also represent the backfiring of an October US waiver on sanctions of Venezuelan oil, which diverted shipments from the South American producer to the US and India, elevating prices for China as shipments dwindled.

The National Iranian Oil Co, China’s commerce ministry and the US Treasury Department did not immediately respond to Reuters requests for comment.

Early last month Iranian sellers told Chinese buyers they were narrowing discounts for December and January deliveries of Iranian Light crude to between $5 and $6 a barrel below dated Brent, five traders who handle the oil or are familiar with the transactions told Reuters.

Those deals had been struck in November at discounts around $10 a barrel, the traders said.

“This is considered as an extensive default and the order to hike prices apparently came from the headquarters in Tehran, as they’re holding back supplies also to the intermediaries,” a China-based trading executive said.

An executive at a Chinese middleman that procures direct from Iran said the OPEC producer was “holding back some shipments”, leading to a “stalemate” between Chinese buyers and Iranian suppliers.

“It’s not clear how things would end,” this executive said. “Let’s wait a bit and see if refineries are willing to accept the new price.”

China has saved billions of dollars buying often deeply discounted oil from sanctioned producers Iran, Venezuela and, more recently, Russia - countries that supply almost 30 per cent of China’s crude imports.

‘TEAPOTS’ SQUEEZED

It is not clear how extensive Iran’s cutbacks to China are. At least one buyer has accepted higher prices: a Shandong-based refiner bought a cargo late last month at discounts between $5.50 and $6.50 on a delivered ex-ship basis, two traders said.

The discounts could narrow further, as the latest offer heard was $4.50, the traders said. Last year’s average discount for Iranian Light, a key grade China buys with a high middle-distillates yield, was about $13, traders say.

“The buyers are still struggling to find a solution as the new prices are too high,” said a Shandong-based buyer. “But since they have limited choices and the Iranian side is very tough, the room for price negotiations is difficult and is not favouring Chinese buyers.”

China’s smaller independent refiners, called “teapots”, have become Tehran’s top clients since first buying Iranian oil in late 2019. They replaced state-run refiners, which stopped dealing with Iran over concerns about falling afoul of US sanctions.

Teapots absorb about 90 per cent of Iran’s total oil exports, usually passed off as oil originating in Malaysia or the United Arab Emirates, trade sources say.

Amid the tussle over prices, Iran’s overall exports and China’s imports from Iran have fallen.

China imported about 1.18 million barrels per day (bpd) of Iranian oil last month, down from 1.22 million bpd in November and 23 per cent off October’s record 1.53 million bpd, tanker tracker Vortexa Analytics reckons.

That represents the bulk of Iran’s global seaborne crude exports, which another tracker, Kpler, estimates at 1.23 million bpd for December, down from 1.52 million bpd in November. Floating storage off Iran and nearby waters rose by about 2 million barrels to 15.5 million barrels over the past week, Kpler says.

“The Iranians want to play catch-up in prices with (Russia’s) ESPO. But they don’t fully realise the extent of sanctions on Iranian oil is different from that on Russian,” said a trading manager at an independent refiner.

Washington has sanctioned more than 180 people and entities related to Iran’s petroleum and petrochemical sectors since 2021, identifying 40 vessels as blocked property of the sanctioned entities.

The main restrictions on Russian oil have been a $60-a-barrel price cap imposed in December 2022 by the US and its allies, aiming to punish Moscow over its invasion of Ukraine. Major buyer India has mostly paid above $60 for Russian oil, hitting $85.42 in November, the highest since the Group of Seven industrial powers imposed the cap.

https://thefinancialexpress.com.bd/world/irans-oil-trade-with-china-stalls-as-tehran-demands-higher-prices

Elevation Updates and Upcoming 2024 Milestones

 Single Agent:

  • Elevation Oncology plans to provide an update from its ongoing Phase 1 trial in mid-2024, with additional data expected in the first half of 2025.
  • In June 2023, Elevation Oncology's partner, CSPC Pharmaceutical Group Limited, presented initial clinical data for SYSA1801 (EO-3021) from their ongoing Phase 1 dose escalation and expansion study in China. Initial data showed promising signs of efficacy, including a 47.1% overall response rate (ORR) in patients with resistant/refractory gastric cancer expressing Claudin 18.2, with a well-tolerated safety profile.
  • In August 2023, Elevation Oncology began enrolling patients in an open-label, multi-center, dose escalation and expansion Phase 1 clinical trial (NCT05980416), designed to evaluate the safety, tolerability and preliminary anti-tumor activity of EO-3021.

Combination:

  • Elevation Oncology plans to expand its clinical development program to evaluate EO-3021 in combination. The Company believes a combination approach has the potential to offer optimal outcomes to patients, particularly in the gastric cancer setting, and plans to explore combination strategies with both immunotherapy and targeted agents. Elevation Oncology expects to share details on its planned Phase 1 combination study in the first half of 2024.

HER3-ADC: Elevation Oncology's second program is a differentiated HER3-targeting ADC. HER3 is a well-validated ADC target, which is overexpressed across solid tumors and often associated with poor outcomes. There are currently no HER3-targeted ADC agents approved for the treatment of cancer.

  • Elevation Oncology is currently evaluating its HER3-ADC program and plans to nominate a development candidate in 2024.

Financial Guidance

Elevation Oncology expects that its cash, cash equivalents and marketable securities as of September 30, 2023, will be sufficient to fund its current operations into the second half of 2025.

https://www.prnewswire.com/news-releases/elevation-oncology-announces-program-updates-and-upcoming-2024-milestones-302026858.html

Lifecore Expands Relation with Existing Long-Term Customer Via Commercial Arrangements

 Lifecore enters into commercial agreements with Alcon that include new 8-year commercial manufacturing arrangements

Lifecore receives amendment and waiver for its term debt facility with Alcon and BMO

https://www.globenewswire.com/news-release/2024/01/05/2804602/23089/en/Lifecore-Biomedical-Expands-Relationship-with-Existing-Long-Term-Customer-Through-a-Series-of-Commercial-Arrangements.html

Late M&A bonanza stokes healthcare dealmakers ahead of JPMorgan conference

Healthcare dealmakers are making their way to San Francisco for a major industry conference, optimistic that more deals are in the offing after a wave of biotech company takeovers at the end of last year.

Kicking-off on Monday, the four-day JPMorgan Healthcare Conference is expected by organizers to attract over 8,000 people, including delegations from the world’s largest drugmakers.

Last month alone, drugmakers including AbbVie, Bristol Myers Squibb and AstraZeneca announced roughly $25 billion worth of U.S.-listed biotech deals, according to data provider LSEG Deals Intelligence.

Overall, global M&A activity in the healthcare sector grew 8% on an annual basis to $365 billion in 2023, lagging the previous five-year average spending of $432 billion, LSEG calculated.

“We've had an uptick of M&A recently, we're seeing stocks rebound with the market recovery and interest rates lowering,” said JPMorgan global head of healthcare investment banking Mike Gaito, who will interview CEO Jamie Dimon on the opening day. “People are open for business.”

Among the hottest topics will be the wildly popular weight-loss drugs revolutionizing the fight against obesity that have established Eli Lilly and Novo Nordisk as two of the world's most valuable companies.

Analysts forecast current drugs and other obesity treatments in development could garner $100 billion a year by the end of the decade.

Gaito said other companies are feeling the pressure to get into the space, and those that are want to be able to offer other treatment options.

Two deals epitomizing this in 2023 were Roche’s $2.7 billion acquisition of Carmot Therapeutics and Eli Lilly’s takeover of Versanis Bio for up to $1.93 billion, which strengthened the Mounjaro maker’s pipeline of obesity drugs.

Other themes will range from regulation and antitrust to the financing environment and possible effects of the 2024 U.S. presidential election on the industry, participants said.

WALL STREET RALLY

The conference comes after a blistering Wall Street rally in recent weeks, propelled by expectations the Federal Reserve will cut interest rates this year.

Biotech companies were among the beneficiaries. The SPDR S&P Biotech ETF, a gauge of biotech industry performance, was up more than 18% in December.

The benchmark U.S. 10-year Treasury note dropped by nearly 50 points last month, easing financing costs for acquirers.

"We expect the macro environment, including how people are thinking about sector growth, interest rates and the labor dynamic, to be top of mind (at the conference),” said Ali Satvat, a partner at private equity firm KKR.

The buying spree by drugmakers late last year was part of their strategy to help offset expected revenue declines as patents on blockbuster therapies expire. AbbVie, already facing biosimilar competition for its cash cow Humira, and Bristol Myers collectively spent roughly $35 billion in deals to bolster their neurology and oncology franchises.

The annual conference at the Westin St. Francis Hotel in San Francisco will include smaller drugmakers and companies from all corners of the healthcare industry, such as health insurers and medical device firms.

Investors will meet companies in public and private settings.

“It’s an opportunity to actually sit in a small group, with investors but also with some corporate people, and meet with the managements to ask questions directly and really gather a lot of competitive intelligence,” said investment firm Perceptive Advisors managing director Doug Giordano.

After a slow 2023, private equity firms will be looking for investment opportunities for a record level of $2.59 trillion of unspent cash. They'll also be searching for buyers. "There is a very significant backlog of private equity companies that will come out for sale in 2024," said Devin O’Reilly, a partner at buyout firm Bain Capital.

Dealmakers from JPMorgan’s rival investment banks and law firms also will be in town to win new business from the over 400 healthcare companies expected to attend.

Competitors set up their own headquarters in hotels near the conference and lure clients by offering better catering and amenities than the official hosts.

Latham & Watkins M&A partner Charles Ruck said you could have fun ranking "who has got better food at their mini conferences around and compare it to the JPMorgan.” 

 https://www.marketscreener.com/quote/stock/JPMORGAN-CHASE-CO-37468997/news/Late-M-A-bonanza-stokes-healthcare-dealmakers-ahead-of-JPMorgan-conference-45680759/