Sens. Rand Paul (R-KY) and Cory Booker (D-NJ) (Olivier Douliery/Sipa USA/Sipa via AP Images)

Un­like­ly duo leads bi­par­ti­san Sen­ate bill to ex­pand Right to Try in­to MD­MA and psilo­cy­bin

Sens. Cory Book­er (D-NJ) and Rand Paul (R-KY) like­ly don’t share much in com­mon when it comes to pol­i­cy views, but that didn’t stop the pair from in­tro­duc­ing a new bill this week that would al­ter the con­tro­ver­sial Right to Try Act to al­low ter­mi­nal­ly ill pa­tients to ac­cess Sched­ule I drugs af­ter a Phase I tri­al has been com­plet­ed.

Build­ing off its pre­de­ces­sor, the bill would skirt around the FDA’s ex­pand­ed ac­cess pro­gram and the DEA’s reg­u­la­tions around con­trolled sub­stances, and al­low pa­tients to po­ten­tial­ly ac­cess Sched­ule I drugs — like MD­MA and psilo­cy­bin — much more eas­i­ly, as the sen­a­tors say these drugs “have shown ex­cep­tion­al promise in treat­ing a va­ri­ety of men­tal health con­di­tions, in­clud­ing sui­ci­dal de­pres­sion, anx­i­ety, and PTSD.”

But as with Right to Try, the FDA is not the bar­ri­er that many as­sume it to be when it comes to ac­cess­ing in­ves­ti­ga­tion­al drugs. The agency signs off on more than 99% of all ex­pand­ed ac­cess re­quests it re­ceives. It al­so re­mains un­clear how many have at­tempt­ed and failed to gain ac­cess to Sched­ule I sub­stances out­side of clin­i­cal tri­als or FDA’s ex­pand­ed ac­cess pro­gram.

Al­i­son Bate­man-House

Al­i­son Bate­man-House, as­sis­tant pro­fes­sor at New York Uni­ver­si­ty and an ex­pert on ex­pand­ed ac­cess, ex­plained to End­points News how this bill builds on oth­er state ex­pan­sions of RTT leg­is­la­tion, even as any doc­tor with the ap­pro­pri­ate li­cens­ing can pre­scribe con­trolled sub­stances.

“This is not an at­tack on the FDA, but just on reg­u­la­tions in gen­er­al, as they don’t care who’s reg­u­lat­ing when it comes to bar­ri­ers to ac­cess,” Bate­man-House said.

But she said she has heard that there’s been a lot of in­ter­est from pa­tients around clin­i­cal tri­als test­ing MD­MA and psilo­cy­bin, al­though some of the larg­er play­ers in this space al­ready have ex­pand­ed ac­cess pro­grams set up.

The Wis­con­sin-based Usona In­sti­tute, for in­stance, pro­vides cGMP psilo­cy­bin for clin­i­cal stud­ies and has a process set up where­by el­i­gi­ble aca­d­e­m­ic re­searchers can ac­cess the sup­plies to ad­vance sci­en­tif­ic and med­ical un­der­stand­ing of psy­che­delics.

“Re­cent stud­ies sug­gest that MD­MA and psilo­cy­bin could rep­re­sent an enor­mous ad­vance­ment in men­tal health and psy­chophar­ma­col­o­gy,” Book­er said in a state­ment. “Un­for­tu­nate­ly, many el­i­gi­ble pa­tients who ur­gent­ly need care do not cur­rent­ly have ac­cess to these promis­ing ther­a­pies.”

Com­pan­ion leg­is­la­tion will be in­tro­duced in the House by Reps. Earl Blu­me­nauer (D-OR) and Nan­cy Mace (R-SC).

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

‘Catchy’ de­sign tops big ad buys on­line for grab­bing on­col­o­gists’ at­ten­tion — sur­vey

The cancer drug ads that get oncologists’ attention online are informative and use clear, eye-catching designs. That’s ZoomRx’s assessment in its most recent tracking survey, and while not necessarily surprising, the details in the research do break a few common misconceptions.

One of those is frequency, also known as the number of impressions an ad gets. No matter how many times oncologists saw a particular cancer drug ad, effectiveness prevailed in the survey across five drug brands. ZoomRx measured effectiveness as a combination of most attention-getting, relevant information and improved perception as reported by the doctors.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

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Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.