Sree Kant, BAKX Therapeutics CEO

Months af­ter mas­sive Ipsen deal, BAKX Ther­a­peu­tics nets more cash for its 'Thel­ma and Louise' ap­proach to can­cer cell death

BAKX Ther­a­peu­tics emerged from stealth in a big way back in Ju­ly, strik­ing an $852 mil­lion deal with Ipsen for its lead can­cer can­di­date, a small mol­e­cule de­signed to ac­ti­vate the body’s nat­ur­al process for pro­grammed cell death. And Ipsen’s putting a bit more cash in the com­pa­ny’s cof­fers to see that pro­gram in­to the clin­ic.

CEO Sree Kant un­veiled a $25 mil­lion Se­ries A round on Thurs­day, led by AB Mag­ni­tude Ven­tures Group with a hand from Ipsen and Sher­pa Health­care Part­ners. The funds will be used to ad­vance the com­pa­ny’s BAKX ac­ti­va­tor pro­gram, which traces back to pi­o­neer­ing work around apop­to­sis by the Dana-Far­ber Can­cer In­sti­tute’s Loren Walen­sky and Al­bert Ein­stein Col­lege of Med­i­cine’s Evri­pidis Ga­vathi­o­tis.

Apop­to­sis is a form of pro­grammed cell death used by the body to get rid of un­need­ed or ab­nor­mal cells. But some can­cer cells are able to evade this mech­a­nism, mul­ti­ply­ing out of con­trol. Com­pa­nies like BAKX are look­ing to re­store that bar­ri­er, in­duc­ing can­cer cell death while spar­ing healthy cells.

Most oth­er ex­per­i­men­tal ther­a­pies go af­ter pro-sur­vival pro­teins called Bcl-2, Bcl-xl and Mcl-1. This strat­e­gy, to “in­hib­it the in­hibitor,” has been ef­fec­tive in cer­tain types of leukemia, with one drug, Ab­b­Vie and Genen­tech’s Ven­clex­ta, se­cur­ing ap­proval back in 2016. But as is of­ten the case with can­cer ther­a­pies, the de­vel­op­ment of re­sis­tance pos­es a chal­lenge.

In­stead, BAKX is tar­get­ing pro-apop­tot­ic pro­teins, such as BAK and BAX, which are down­stream of Bcl-2, Bcl-xl and Mcl-1. By di­rect­ly ac­ti­vat­ing these pro­teins, sci­en­tists be­lieve they can dri­ve apop­to­sis and de­stroy tu­mor cells.

Kant com­pared the ap­proach to Thel­ma and Louise at the edge of the cliff. You could dri­ve the can­cer cell off the cliff in one of two ways: You can let off the brakes, or press the ac­cel­er­a­tor. While pro-sur­vival pro­tein in­hibitors take the brakes off, BAKX is press­ing the ac­cel­er­a­tor.

The biotech’s lead can­di­date, a small mol­e­cule ac­ti­va­tor of BAX called BKX-001, is ex­pect­ed to en­ter the clin­ic in 18 to 24 months, Kant said con­ser­v­a­tive­ly.

“Hope­ful­ly we can do it much, much ear­li­er,” he added.

Ipsen put down $14.5 mil­lion up­front and $837.5 mil­lion in biobucks to col­lab­o­rate on the can­di­date back in Ju­ly. While the part­ners are hop­ing to go af­ter leukemia, lym­phoma and sol­id tu­mors, Kant says they’ll like­ly fo­cus on hema­tol­ogy first. BAKX al­so has two dis­cov­ery pro­grams for undis­closed tar­gets up its sleeve.

“I wasn’t look­ing at sci­ence which would be a good sto­ry I can sell to the in­vestors,” Kant said. “The on­ly thing I’m think­ing of is sci­ence that I can make a drug with. So if my daugh­ter asks me what do you do for a liv­ing, I can tell her I make drugs.”

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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CEO Lex Rovner (64x Bio)

A George Church spin­out fight­ing the vi­ral vec­tor bot­tle­neck in cell and gene ther­a­py lands $55M

A synthetic biology company spun out of George Church’s lab is set to tackle the gene therapy manufacturing bottleneck, and it just landed $55 million in a Series A financing round to do so.

64x Bio comes out of the Harvard Department of Genetics. CEO Lex Rovner and her team — which right now, sits around 10 people — are looking to tackle a key hurdle for major companies: manufacturing cell and gene therapies.