Author: Randy Chase

So, how much do you know about bispecific therapy?

Can you even offer a passing definition?

Well, read on dear reader….. and learn enough to impress your colleagues!

Why is knowing about bispecific therapies even remotely important?

Although they have been around for more than a decade, bispecifics are back and making big news in disease management. Currently, there are over 100 bispecifics in clinical development. AstraZeneca, BioNTech and Summit Therapeutics are just a few pharma with pipeline products. KOLs believe that bispecifics are the next new thing in immunotherapy with promise to even surpass the pioneering performance of monoclonal antibody therapy Keytruda.

Here’s the definition that you need to know to sound well informed……

Bispecific antibodies are engineered to bind to cancer cells and healthy immune system cells (T-cells) at the same time, activating the healthy immune system cells to recognize and destroy cancerous cells. 

The article below does a good job of filling in more detail to assuage your insatiable curiosity.

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With Myriad Recent Approvals in Cancer, The Era of Bispecifics Is Here

 As companies roll out data showing the power and improved safety profile of antibodies that target two antigens, analysts say the class could overtake monoclonal antibody Keytruda as the “immunotherapy backbone” of solid tumor treatment.

CLICK HERE to read the full article

This new year should be verrrrry interesting…… especially with respect to the FDA and a host of changes that are likely to be implemented in the agency. One area that is likely to come under the microscope is the FDA Accelerated Approval Pathway.

In short, the pathway is a shortcut to approval of a new therapy. On its surface, the rationale behind accelerated approval makes a lot of sense. Why delay making a therapy with great ‘promise’ available, especially a life-saving therapy, to reach desperate patients as soon as possible.

As noted in the article below, the pathway was implemented in 1992 in response to the HIV/AIDS epidemic, accelerated approval enables drugs to be approved based on a surrogate endpoint that is reasonably likely to predict clinical benefit. These FDA approvals allow pharmaceutical companies to begin selling these drugs on the condition that they conduct confirmatory trials. Since its inception, the pathway has helped bring nearly 300 new drugs to the market, many for conditions with high unmet need.

Well, as with most things, perfection in pharmaceuticals is also a rare commodity. Fortunately, the number of therapies that originally received accelerated approval, only to be later yanked off the market due to ‘unforeseen consequences’ (sometimes fatal), have been relatively few. None the less, only a few ‘oops’ beg the question…. Are even a few deaths or other serious adverse events worth the risks?

The article below is a good read that adds perspective to the question of accelerated approvals and their future in the FDA approval process.

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5 Accelerated Approvals Gone Wrong

CLICK HERE to read the full article

The year 2024 was a very busy year for specialty pharmacy. So many SP therapies were approved by the FDA that we are still catching up on all the announcements.  

What we did NOT see in 2024 was much activity on the business front….. especially SP acquisitions, major expansions, etc. However, days ago, perhaps the biggest story of the year hit the ether….. Walgreens (WAGS) is facing a serious reversal of fortunes to the tune of $billions$ of dollars. The numbers are jaw dropping….. about 10 years ago Walgreens’ market cap was more than $100 billion. They ended the year with a valuation of only $8 billion.

We’ve watched with curiosity the remaking of WAGS over the past decade and much of that transformation came with $billion$ dollar price tags. That spending spree has cost WAGS dearly leading to discussions with at least one private equity firm to form a consortium to take WAGS private.  

Current WAGS management has been aggressively cleaning shop including closing underperforming retail stores and whole divisions such as VillageMD. As is typical in these deals, the investors will continue to slash operating costs and sell off assets that are not accretive or simply don’t ‘fit’ in the new company vision.  

What might WAGS look like in the wake of a going private?

Only the profitable retail locations will survive.

WAGS will actively partner with third parties to hedge financial exposure.

WAGS will continue to pursue deals with hospitals and health systems, one of the strategies that seem to have been successful over recent years.

WAGS will continue to compete in specialty pharmacy.

Succeeding in specialty pharmacy may not be as easy as it might look.  

The acquisition of Shields Health was integral to structure deals with hospitals and health systems around specialty pharmacy. But, once that intellectual property has been transferred, will clients feel the need for those contracts to be renewed?  

There are three articles worth reading to get a better lay of the financial landscape that WAGS needs to traverse in coming days. 

CLICK HERE to read the Forbes article  Walgreens in talks to go private

CLICK HERE to read the Axios article    Sycamore Walgreens take private partner

CLICK HERE to read the Axios article    Walgreens Rocky Road

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the early press releases rarely specify the specialty pharmacy(ies) selected as the designated partner(s).

Here are seven LD deals that have been publicly confirmed subsequent to their approvals.

Orsini to Distribute Furoscix  

Orsini has been selected by SC Pharmaceuticals to distribute Furoscix (furosemide injection), an Infused diuretic for Congestive Heart Failure indicated for the treatment of congestion due to fluid overload in adults. Furoscix is available as a subcutaneous, single-use therapy that can be administered at home by patients and caregivers.

Orsini to Distribute Sajazir

Orsini has been chosen by Cycle Pharmaceuticals to distribute Sajazir (icatibant) Injection, a subcutaneous treatment for acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older. 

CareMed to Distribute Leqembi

CareMed, a BrightSpring Health Services company, has been selected by Eisai to be the national specialty pharmacy provider for Leqembi, indicated for Alzheimer’s disease.

Onco360 to Distribute Leqembi

Onco360 has been selected as a pharmacy partner by Genentech to distribute Itovebi (inavolisib) which is indicated, in combination with palbociclib and fulvestrant, for the treatment of adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy.

Onco360 to Distribute Revuforj

Onco360 has been selected as a national pharmacy partner by Syndax Pharmaceuticals for Revuforj (revumenib). Revuforj is the first and only menin inhibitor approved for the treatment of relapsed or refractory (R/R) acute leukemias with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older.

Orsini to Distribute Casgevy

Orsini has been selected by Vertex Pharmaceuticals Inc to dispense gene-editing therapy Casgevy (exagamglogene autotemcel). Casgevy is a CRISPR/Cas9 gene-edited cell therapy indicated for the treatment of patients aged 12 years and older with (1) sickle cell disease (SCD) with recurrent vaso-occlusive crises or (2) transfusion-dependent β-thalassemia (TDT).

InspiroGene by McKesson to Distribute Casgevy

InspiroGene* has been selected by Vertex Pharmaceuticals Incorporated to expand commercial distribution options for Casgevy (exagamglogene autotemcel). Casgevy is a CRISPR/Cas9 gene-edited cell therapy indicated for the treatment of patients aged 12 years and older with (1) sickle cell disease (SCD) with recurrent vaso-occlusive crises or (2) transfusion-dependent β-thalassemia (TDT).

_*  InspiroGene is a dedicated business unit of McKesson that specifically focuses on the commercialization of cell and gene therapies (CGTs) and operates in conjunction with business units Biologics by McKesson Specialty Pharmacy and specialty distributor McKesson Plasma & Biologics. 

…………………….. catching up on FDA approvals

The FDA recently approved a new therapy, Hympavzi (marstacimab-hncq) from Pfizer Inc., indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors.

This approval is yet another, cutting edge development in the hemophilia category in the past of year+. Hympavzi is the first of its kind and works by targeting a protein in the blood clotting process.

For decades, clotting factor products merely replaced clotting factor. Hympavzi works by reducing the amount of tissue factor pathway inhibitor, a naturally occurring anticoagulation protein. As such, it increases the amount of thrombin, an enzyme that is critical in blood clotting. Ultimately this is approach is expected to reduce the frequency of bleeding episodes.  

Hympavzi is a once-weekly subcutaneous injection and is supplied in pre-filled auto-injector pens.

CLICK HERE to access prescribing information

Pfizer set a wholesale acquisition cost (WAC) at $795,600 for Hympavzi. 

By comparison to other recent hemophilia breakthrough therapies, Pfizer charges $3.5 million for its Beqvez product and matches the price for Hemgenix , a hemophilia B gene therapy.

The company announced that Hympavzi would launch through Orsini Specialty Pharmacy limited distribution.

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FDA Approves New Treatment for Hemophilia A or B

Product is First Non-Factor and Once-Weekly Treatment for Hemophilia B

CLICK HERE to access the press release 

If you work in specialty pharmacy it is a safe bet that you are familiar with pharmacy accreditation. If you are familiar with accreditation you understand that accreditation is a comprehensive approach to measure specialty pharmacy performance against specific standards. And, if you are that familiar with accreditation, you almost assuredly are familiar with URAC, one of the leading independent accrediting organizations in the US.

URAC recently released a report that summarizes performance among its numerous participating specialty pharmacies with a five year lookback. The report focuses on key metrics that are valuable for specialty pharmacies to gauge their own performance against competing specialty pharmacies in the marketplace. Payers and even patients are increasingly attuned to such performance metrics when seeking to do business with an organization that has the health, safety, and financial fortunes in their hands.

Some of the leading metrics cited in the new report include;

• Quality monitoring enhancement
• Fostering industry standards
• Improvement in call center performance
• Dispensing accuracy enhancements
• Challenges in distribution accuracy
• Turnaround time trends
• ….. and more.

The report, therefore, should be mandatory reading for every specialty pharmacy.

CLICK HERE to access the URAC Five-Year Specialty Pharmacy Report: Making a Difference With Measurement

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URAC Releases Specialty Pharmacy Report

• Over the five-year period, URAC-accredited pharmacies responded to patient calls within 30 seconds 75% of the time, improving to 86% by 2023.

CLICK HERE to read the article

…………………………catching up on FDA approvals

The FDA recently approved an ORAL therapy, Itovebi (inavolisib) from Genentech, Inc. Itovebi is indicated in combination with palbociclib and fulvestrant for the treatment locally advanced or metastatic breast cancer in adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy.

Itovebi is a type of targeted therapy medicine called a PI3K inhibitor. Previously, Piqray (alpelisib) was the only PI3K inhibitor approved to treat breast cancer with a PIK3CA mutation.  Approximately 70% of all breast cancers are hormone-receptor positive, HER2-negative.  PIK3CA is the most commonly mutated gene in hormone-receptor positive, HER2-negative breast cancer, with approximately 40% of patients harboring this mutation. 

Genentech said that Itovebi will launch at a price of $22,867 for a 28-day cycle.

Distribution details were not announced. Although it comes with a relatively high cost, Good Rx shows current availability at a large number of retail pharmacies including CVS, Walgreens, Walmart, etc. 

CLICK HERE to access prescribing information

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FDA approves inavolisib with palbociclib and fulvestrant for endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative, advanced breast cancer

CLICK HERE to read the full press release

Quiz me this……………

Would you prefer to have your medication administered intravenously or in pill form?

Did it take more than a nano second to answer that question?

Probably not.

The article below may have some good news for the vast number of patients that deal with the burdens of an infused course of therapy.  As the article below points out, infusions are inconvenient or even inaccessible to patients….. and, complications often arise not from the active drug, but the infusion itself. “Turning these drugs into pills could be transformative.”

But is it doable?  

Researchers say ‘YES’. They are actively working to solve a big hurdle….. ‘bioavailability’….. or how to make oral medications more readily dissolvable in the bloodstream with maximum absorption. (Ever try to mix a vinegrette dressing? Ya got to make the oil combine with the vinegar or water.) The article explains how new technology is being developed to achieve exactly that….. think routinely breaking the oil vs. water barrier.

Are you familiar with the term ‘sol-moiety’? Existing drugs are now being tested in the lab to achieve bioavailability using this technique….. and results have been spectacular with bioavailability increases from nearly zero to 100% being observed. Proof-of-concept is no longer a hurdle.

Read the full article for more details and perhaps a glimpse of the future.

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New strategy could turn intravenous medicines into pills

CLICK HERE to access the full article

Among the many earth-moving changes on the payer / PBM battlefield was last year’s announcement that Blue Shield of California was breaking away from its relationship with CVS’ Caremark and creating a very different model that would include multiple partners. According to the company, partners include Amazon Pharmacy, Abarca, Mark Cuban Cost Plus Drug Company, and Prime Therapeutics, with Caremark still hanging onto specialty pharmacies.

But wait….. BSofCA just announced another big change as part of its ‘Pharmacy Care Reimagined’ model that will further shake up the marketplace opening the door for other payers to emulate.

In short, BSofCA is dethroning Humira from what has been a sacrosanct position at the pinnacle of its specialty drug formulary. Instead, “Blue Shield is teaming up with drugmaker Fresenius Kabi and Evio Pharmacy Solutions to purchase a biosimilar for Humira at a transparent net price of $525 per monthly dose.” That price is significantly below the market reported net price of $2,100.

Moreover, Blue Shield confirmed that members will pay a $0 copay for the biosimilar. This makes the shift a big win financially for both the health plan….. and members. It is also believed that the no cost to member option will measurably boost persistence and improve overall health outcomes.

Details on how exactly Blue Shield will actually adjudicate and ‘pay’ for the meds were not announced. That, in itself, will be noteworthy. This should be fun to follow.

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Blue Shield of California inks deal to buy Humira biosimilar directly from manufacturer

CLICK HERE to read the article

……………………. catching up on FDA Approvals

The FDA recently approved a new infused therapy, Niktimvo (axatilimab-csfr) from Incyte Corporation and Syndax Pharmaceuticals, indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. 

Graft-versus-host disease is a leading cause of death from bone marrow transplants, which are used to treat certain blood cancers, solid tumors and immune disorders. It is estimated that approximately a third of those who receive bone marrow transplants experience GVHD.

Niktimvo doesn’t work like its predecessors which include Incyte’s Jakafi and Sanofi’s Rezurock. Niktimvo is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody that targets and blocks a protein involved in the development and survival of certain kinds of white blood cells.

Analysts suggest that Niktimvo will launch with a list price of $21,500 / month.

The company did not announce details for distribution.

CLICK HERE to access prescribing information

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FDA approves axatilimab-csfr for chronic graft-versus-host disease

CLICK HERE to access the FDA press release