The first pa­tient has been treat­ed un­der the con­tro­ver­sial 'right to try' law

A pa­tient di­ag­nosed with an ag­gres­sive form of brain can­cer be­came the first per­son in the US to ac­cess an ex­per­i­men­tal treat­ment un­der Right to Try. Signed in­to law by Pres­i­dent Don­ald Trump in May 2018, it has been tout­ed as a new way to help peo­ple with ter­mi­nal ill­ness­es and few op­tions, al­though un­til now, no pa­tients had ever used it.

ERC-USA and the Uni­ver­si­ty of Cal­i­for­nia, Irvine ini­ti­at­ed treat­ment with the com­pa­ny’s in­ves­ti­ga­tion­al com­pound ERC-1671 — known as Glio­vac in Eu­rope — and which is in Phase 2 clin­i­cal tri­als in the US. The pa­tient’s treat­ment with ERC-1671 be­gan at the uni­ver­si­ty in late No­vem­ber 2018.

The pa­tient re­sort­ed to Right to Try af­ter fail­ing to qual­i­fy for en­roll­ment in the on­go­ing tri­al. But rather than work around the agency that may even­tu­al­ly ap­prove the ex­per­i­men­tal treat­ment, the com­pa­ny said in Au­gust that it in­formed the FDA that it in­tend­ed to make the ex­per­i­men­tal treat­ment avail­able to this one pa­tient. FDA ac­knowl­edged ac­cep­tance of the com­pa­ny’s no­ti­fi­ca­tion on 13 Ju­ly, ERC said.

Oth­er com­pa­nies, in­clud­ing Ther­a­peu­tics So­lu­tions In­ter­na­tion­al, have an­nounced their in­ten­tions to use Right to Try, though ac­cord­ing to Gold­wa­ter In­sti­tute, no oth­er pa­tients have yet to use the new law.

The law has proven to be a thorn in the side of FDA, which has said it will work to im­ple­ment it in a man­ner con­sis­tent with con­gres­sion­al in­tent and with FDA’s pub­lic health mis­sion, but the agency still has its own process for help­ing ter­mi­nal­ly ill pa­tients re­ceive ex­per­i­men­tal treat­ments, known as ex­pand­ed ac­cess. FDA grants about 99% of the ex­pand­ed ac­cess re­quests it re­ceives.

Ac­cord­ing to Bio­Cen­tu­ry, FDA’s On­col­o­gy Cen­ter of Ex­cel­lence is al­so work­ing on a new ini­tia­tive, known as Pro­ject Fa­cil­i­tate, un­der which the agency will pro­vide a tele­phone num­ber that pa­tients and physi­cians seek­ing ex­pand­ed ac­cess to an ex­per­i­men­tal treat­ment can call. FDA staff will an­swer calls and fill out the form re­quired to ap­ply for a sin­gle-pa­tient IND re­quest. The pa­per­work will be for­ward­ed to the man­u­fac­tur­er. A pi­lot ver­sion of this ini­tia­tive is ex­pect­ed to launch in the first half of 2019.


First pub­lished here and syn­di­cat­ed in part­ner­ship with RAPS.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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Sean Nolan and RA Session II

Less than 3 months af­ter launch, the AveX­is crew’s Taysha raise $95M Se­ries B. Is an IPO next?

The old AveXis team is moving quickly in Dallas.

Three months ago, they launched Taysha with $30 million in Series A funding and a pipeline of gene therapies out of UT Southwestern. Now, they’ve announced an oversubscribed $95 million Series B. And the biotech is declining all interview requests on the news, the kind of broad silence that can indicate an IPO is in the pipeline.

Biotechs, including those relatively fresh off launch, have been going public at a frenzy since the pandemic began. Investors have showed a willingness to put upwards of $200 million to companies that have yet to bring a drug into the clinic. Still, if Taysha were to go public in the near future, it would be perhaps the shortest path from launch to IPO in recent biotech memory.

UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Mod­er­na CEO Stéphane Ban­cel out­lines a prospec­tive moth­er­lode of Covid-19 vac­cine rev­enue — will a back­lash fol­low?

Moderna shows no sign of slowing down, or turning charitable when it comes to pricing supplies of its Covid-19 vaccine.

One of the leaders in the Phase III race to get a Covid-19 vaccine across the finish line in record time, Moderna says it’s on track to complete enrollment in one of the most avidly watched studies in the world next month. And the biotech has already banked some $400 million in deposits for vaccine supply as it works through negotiations with countries around the world — as CEO Stéphane Bancel sets out to hire a commercial team.

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My­ovant lands a fresh $200M loan as FDA mar­ket­ing de­ci­sion looms; Amarin goes it alone in Eu­rope

Myovant is getting ready to roll out its commercial operations to back relugolix, now under FDA review for prostate cancer.

The startup has added a fresh $200 million in support from Sumitomo Dainippon Pharma, which controls a majority of the stock $MYOV. Sumitomo is handing the cash over as a loan, bringing its total to $600 million. Myovant — which is gearing up for a showdown with AbbVie — has also filed an NDA to sell relugolix for uterine fibroids and recently posted positive late-stage data for endometriosis.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Covid-19 roundup: J&J and BAR­DA agree to $1 bil­lion for 100 mil­lion dos­es; Plas­ma re­duces mor­tal­i­ty by 50% — re­ports

J&J has become the latest vaccine developer to agree to supply BARDA with doses of their Covid-19 vaccine, signing an agreement that will give the government 100 million doses in exchange for $1 billion in funding.

The agreement, similar to those signed by Novavax, Sanofi and AstraZeneca-Oxford, provides funding not only for individual doses but to help J&J ramp up manufacturing. Pfizer, by contrast, received $1.95 billion for the doses alone. Still, if one looked at each agreement as purchase amounts, J&J’s deal would be $10 per dose, slotting in between Novavax’s $16 per dose and AstraZeneca’s $4 per dose.

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Ver­sant de­buts Ridge­line's start­up #4, armed with $30M and al­ter­na­tive TCR cell ther­a­pies for sol­id tu­mors

For all the iterations and advances in TCR therapies for cancer, any experimental treatments involving T cell receptors share one trait: By definition, they only recognize antigens presented as peptides on the major histocompatibility complex (MHC) on cells.

Versant reckons it’s time to expand the arsenal. With $30 million in initial funding, its Ridgeline Discovery Engine in Switzerland has been working on a non-peptidic approach that it says has tumor-agnostic potential, especially in solid tumors. They’ve named it Matterhorn, after a Swiss mountain as they did with the three other companies that have emerged from the Basel-based incubator.

CF Foun­da­tion, Long­wood team on new in­cu­ba­tor for com­pa­nies with cut­ting-edge CF treat­ments

Nine months after launching a $500 million hunt for a cure for cystic fibrosis, the Cystic Fibrosis Foundation said it will use a portion of those funds to do something it has never done before: help launch new companies.

The CF Foundation, whose venture philanthropy efforts helped fund Vertex’s line of powerful CF drugs, is teaming with Longwood Fund to create a CF incubator. The incubator will identify new companies with platforms or technologies that can be applied in the rare genetic condition. The partners can then finance early development in exchange for a commitment from the companies to focus on applications in cystic fibrosis.