Bill Gates has a $100M and a 5-point strat­e­gy to end 15 years of fail­ure in Alzheimer’s R&D

Bill Gates is go­ing af­ter Alzheimer’s.

The soft­ware bil­lion­aire is in­vest­ing $50 mil­lion of his own mon­ey — not from the Gates Foun­da­tion — in­to the in­dus­try/gov­ern­ment backed De­men­tia Dis­cov­ery Fund, which set out a cou­ple of years ago to back some trans­la­tion­al work on new ap­proach­es to the dis­ease. And he tells Reuters that he’s ear­marked $50 mil­lion more to back new com­pa­nies on his own that have the po­ten­tial to tack­le some fresh ap­proach­es to a dis­ease that has de­feat­ed vir­tu­al­ly every­thing thrown at it in the last 15 years.

Most of the big Phase II­Is have looked to elim­i­nate two pos­si­ble caus­es of Alzheimer’s — amy­loid be­ta and tau — in a crude at­tempt to bend the curve of the dis­ease. But all the piv­otal work by Eli Lil­ly, J&J, Mer­ck and oth­ers has failed bad­ly over the years.

Gates wants to go in some new di­rec­tions.

In an in-depth blog post dis­cussing his in­vest­ment in the fund, which is man­aged by SV Life Sci­ences, Gates out­lined his own 5-part strat­e­gy to tak­ing a fresh ap­proach. His thoughts:

  • We are woe­ful­ly ig­no­rant of how this dis­ease de­vel­ops and what may be dri­ving it. Why are blacks and Lati­nos more like­ly to de­vel­op Alzheimer’s? No one knows. So it’s time to go back and do the ba­sic re­search to study caus­es and bi­ol­o­gy.
  • Let’s de­vel­op a re­li­able way — per­haps a blood test — to di­ag­nose Alzheimer’s ear­li­er. The on­ly sure-fire way to do it now is through an au­top­sy, which has some ob­vi­ous lim­i­ta­tions in terms of mount­ing clin­i­cal stud­ies.
  • Wouldn’t it be great if tau and amy­loid be­ta worked as a tar­get? (Gates is noth­ing if not sup­port­ive.) Yes, but he wants to fund some new ideas.
  • “If we could de­vel­op a process to pre-qual­i­fy par­tic­i­pants and cre­ate ef­fi­cient reg­istries,” Gates writes, “we could start new tri­als more quick­ly.”
  • Com­pile all the da­ta out there in one plat­form and let re­searchers ex­plore it for new clues to de­vel­op­ing drugs and di­ag­nos­tics. Not sur­pris­ing­ly for the Mi­crosoft founder, that’s Gates’ sweet spot and where he might fo­cus much of his at­ten­tion.

“My back­ground at Mi­crosoft and my (Gates) Foun­da­tion back­ground say to me that a da­ta-dri­ven con­tri­bu­tion might be an area where I can help add some val­ue,” he told Reuters.

Gates is in it for the long haul. He ex­pects it could quite like­ly be a decade or more be­fore some­thing sub­stan­tial comes out of this all. And as a re­sult it makes more sense to in­vest his own mon­ey rather than the foun­da­tion’s. He notes:

I’m mak­ing this in­vest­ment on my own, not through the foun­da­tion. The first Alzheimer’s treat­ments might not come to fruition for an­oth­er decade or more, and they will be very ex­pen­sive at first. Once that day comes, our foun­da­tion might look at how we can ex­pand ac­cess in poor coun­tries.

Bill Gates has one of those leg­endary tech rep­u­ta­tions all biotechs love to be as­so­ci­at­ed with. And Gates-backed biotechs have that ex­tra lus­ter that opens doors, rais­es cash and gets the at­ten­tion of the ma­jor play­ers. The Alzheimer’s R&D groups he will back over the com­ing years have a lot more to gain than his mon­ey.


Im­age: Bill Gates. WEC

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.