Aim­mune wins ap­proval for peanut im­munother­a­py, charges 10k+

The FDA has ap­proved the first treat­ment for peanut al­ler­gy, al­though it comes with some con­tro­ver­sy.

Un­like most of the con­tro­ver­sial drugs that come be­fore the FDA, the bulk of ques­tions don’t cen­ter around this drug’s ef­fec­tive­ness or side ef­fects.

Pal­forzia, de­vel­oped by Aim­mune, has been shown to pre­vent the most se­vere re­ac­tions from ex­po­sure to small amounts of peanuts. In a Phase III tri­al, chil­dren giv­en es­ca­lat­ing dos­es of the drug were then giv­en 600 mg peanut pro­tein — about 2 peanuts — had a 67% chance of avoid­ing a se­vere re­ac­tion. For the place­bo pa­tients, the fig­ure was 4%.

Jayson Dal­las Aim­mune

“The goal — step 1 in terms of ther­a­py — is to be able to treat a child to the point you can say, if that child goes in­to the com­mu­ni­ty and ac­ci­den­tal­ly gets ex­posed to a peanut some­where, they’re not go­ing to have a life-threat­en­ing event,” Aim­mune CEO Jayson Dal­las told End­points News in an in­ter­view at the JP Mor­gan con­fer­ence ear­li­er this month.

“They may have a no re­ac­tion at all, they may have a lit­tle tin­gling, but they’re not go­ing to be at risk.”

The FDA ad­vi­so­ry com­mit­tee point­ed out that this wasn’t ide­al — you can’t eat a peanut but­ter sand­wich and a sig­nif­i­cant num­ber of the pa­tients couldn’t even eat the 2 peanuts  — and not­ed the ad­verse re­ac­tions some had to the pill. But large­ly, they agreed it was a sub­stan­tial step for­ward for a con­di­tion that has seen vir­tu­al­ly no in­no­va­tion.

Crit­ics, though, have point­ed to what’s in the drug: peanuts. Pal­forzia is a form of im­munother­a­py, by which pro­longed ex­po­sure to an al­ler­gen — in this case, peanut pow­der, most­ly but not en­tire­ly equiv­a­lent to what you can find on a su­per­mar­ket shelf — is used to build up re­sis­tance. Some doc­tors have long done this with­out any pre­scribed drug and to some suc­cess. In a 2018 study, re­searchers start­ed 270 pa­tients on 1/2,500th of a peanut and worked 80% of them up to 12 peanuts.

Pal­forzia, op­po­nents say, would take this off-la­bel treat­ment and charge thou­sands of dol­lars for it. An­a­lysts had pro­ject­ed a lit­tle over $4,000 for the price and over $1 bil­lion in peak sales. The ac­tu­al an­nu­al price, Aim­mune an­nounced to­day, will be $10,680.

The In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view al­so point­ed to some of the ad­verse ef­fects pa­tients ex­pe­ri­enced from re­ceiv­ing the drug, and said the long-term ben­e­fit wasn’t clear enough to jus­ti­fy the cost and those ad­verse ef­fects.

Aim­mune has said those risks are to be ex­pect­ed when ex­pos­ing pa­tients to an al­ler­gen. They ar­gued that while doc­tors can pre­scribe pa­tients off-la­bel peanut pow­der, they are ex­posed to law­suits for any re­ac­tion a kid could have to the pow­der. By bring­ing the drug in­to the med­ical sys­tem, they say they give an op­tion for doc­tors to pre­scribe pa­tients with­out mak­ing them­selves li­able.

The drug is ap­proved for chil­dren ages 4 to 17. Aim­mune said they plan to hold pre­sen­ta­tions and work­shops to train 1,300 al­ler­gists on how to ad­min­is­ter the drug.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Hervé Affagard, MaaT Pharma CEO

One year in­to clin­i­cal hold, FDA has more ques­tions about 'pooled' mi­cro­bio­me ther­a­py

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”

Feud be­tween two biotechs left near­ly 12M dos­es of mon­key­pox an­tivi­ral on the ta­ble

As the US embarks on a new delivery strategy to stretch out its thinning supply of monkeypox vaccines, the need for treatments could pick up as cases of the virus rise. And the amount of courses of one potential antiviral, soon to be clinically tested for efficacy in humans, was almost 12 million more than it is today, according to SEC filings.

While not greenlit for treating monkeypox, SIGA’s FDA-approved smallpox antiviral, Tpoxx, can be requested by physicians under an expanded use program. As of Monday, HHS tells Endpoints News it had tapped into more than 15,000 of the 1.7 million courses of Tpoxx that have been stockpiled, but with cases climbing over the past few weeks, demand will likely not peter out in the near future, especially if the vaccine supply runs dry.

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