As­traZeneca gets in on RNA game with Mi­NA re­search col­lab­o­ra­tion

Two months af­ter No­var­tis spent near­ly $10 bil­lion on The Med­i­cines Com­pa­ny’s RNA-based cho­les­terol drug, As­traZeneca will try to co-de­vel­op its own RNA-based meta­bol­ic ther­a­py.

The Eu­ro­pean drug­mak­er has part­nered with Mi­NA Ther­a­peu­tics to de­vel­op small ac­ti­vat­ing RNA (saR­NA) for meta­bol­ic dis­eases. The agree­ment gives As­traZeneca the right to ne­go­ti­ate a li­cens­ing agree­ment af­ter a se­ries of pre­clin­i­cal stud­ies.

Like As­traZeneca’s re­cent $1 bil­lion ex­pan­sion in­to Chi­na, the deal marks an­oth­er ef­fort by the lega­cy phar­ma to keep up with the lat­est tech­nol­o­gy in its es­tab­lished ther­a­peu­tic ar­eas.

The part­ner­ship is the third ma­jor phar­ma col­lab­o­ra­tion for Mi­NA. The biotech part­nered with Japan-based So­sei in a deal that gave Mi­NA $45 mil­lion and So­sei a 25% stake along with an ex­clu­sive op­tion to buy the com­pa­ny for $140 mil­lion, pend­ing the out­come of a Phase I/II tri­al on Mi­NA’s liv­er can­cer drug can­di­date.

The same year, Mi­NA signed an up-to €307 mil­lion deal with Boehringer In­gel­heim for three liv­er fi­bro­sis tar­gets, in­clud­ing at least one NASH drug. So­sei passed on the buy­out op­tion in 2018 amid Phase I tri­al re­sults that showed lit­tle aside from safe­ty and phar­ma­co­ki­net­ics, but they main­tained their 25% stake in the com­pa­ny.

It’s not clear yet which spe­cif­ic in­di­ca­tions As­traZeneca and Mi­NA will fo­cus on. In an in­ter­view with Fierce­Biotech, Mi­NA CEO Robert Habib cit­ed in­clisir­an, the RNA in­ter­fer­ence (RNAi) cho­les­terol drug No­var­tis ac­quired from The Med­i­cines Com­pa­ny, as an ex­am­ple of RNA’s po­ten­tial in meta­bol­ic dis­eases.

“That shows ex­act­ly the pow­er of an RNA ther­a­py of­fer­ing su­pe­ri­or phar­ma­col­o­gy to pa­tients in meta­bol­ic dis­ease,” he said.

Robert Habib

Mi­NA’s saR­NA tech is dis­tinct, though, from the RNAi ther­a­pies of­fered by No­var­tis and Al­ny­lam.

Mi­NA’s pub­lished pa­pers de­scribe small ac­ti­vat­ing RNA as “sim­i­lar to RNA in­ter­fer­ence” in that they both re­ly on short strands of RNA and a pro­tein called Arg­onaute-2. But they do es­sen­tial­ly op­po­site things in the cell. RNAi si­lences genes; it hi­jacks a nat­ur­al cel­lu­lar process and cuts up RNA that ex­press­es a dis­ease-caus­ing pro­tein. saR­NA am­pli­fies; it in­duces what’s called tran­scrip­tion­al elon­ga­tion, help­ing cells cre­ate more of a pro­tein that’s be­ing un­der-ex­pressed.

For ex­am­ple, Mi­NA’s liv­er can­cer drug is de­signed to re­store the ex­pres­sion of CEB­PA, a gene hy­poth­e­sized to al­ter the im­mune sys­tem in the tu­mor mi­croen­vi­ron­ment and make the tu­mor more sus­cep­ti­ble to can­cer drugs.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Ku­ra co-founder heads to Asian mul­ti-na­tion­al as biotech eyes the goal posts for lead drug

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.

FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.

The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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2019 a 'trans­for­ma­tive year' for phar­ma M&A. Is that a good thing?

Big Pharma keeps getting bigger.

Fueled by the mega-mergers between Bristol-Myers Squibb and Celgene and between Allergan and AbbVie, the industry last year saw $350 billion worth of M&A, according to the new year-end report from the consultants at PwC.  That’s a more than 50% increase on 2018.

“I kind of look at 2019 as a transformational year,” report author Glen Hunzinger told Endpoints News. 

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Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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